Mucopolysaccharidoses (MPS) are a group of very rare disorders, also known as orphan diseases. They belong to the group of lysosomal storage diseases which are caused by a deficiency of one of the enzymes involved in the degradation of mucopolysaccharides (the acid glycosaminoglycans or GAGs). The enzymes are coded by genes which produce deficient gene products due to gene variants in each of the two gene-alleles.

Children of two carriers as parents have a 25% risk to suffer from MPS. For many families, the birth of the first affected child is a shock and a disaster. The disease is continuously progressing, and life spans are dramatically decreased without therapy. As a result, extensive efforts are put into the cure of these fatal disorders.

Enzymes are relatively small proteins, produced in the endoplasmatic reticulum of each cell. Before reaching the locus of their function, the lysosomes, additional modifications with special sugars are performed in the Golgi apparatus (glycosylation). Via mannose-6-phosphate marker, they connect to the mannose-6-phosphat receptor on the lysosomal membrane and can reach the final locus of their function. In the lysosomes, enzymes degrade the GAG chains into the smallest molecules for recycling or excretion. Any disturbance in this process leads to the accumulation of non-degraded material, which affects many other cell functions such as homeostasis, calcium metabolism, accelerates apoptosis and induces inflammation processes.

As lysosomes are ubiquitous, any disturbance leads to storage in many different tissues and organs. MPSs are a good example for chronic progressive multi-systemic disorders. The best theoretical option for treatment of any patient is to supplement the missing enzyme which could reach any organ via blood flow and get inside the lysosomes continuing the interrupted degradation processes.

The enzymes are ubiquitous and have some tissue specific compositions. Enzymes produced in the different cells and tissues have their own characteristics and are available on site. The production of recombinant enzymes means that the artificial glycosylation is created in a uniform composition for intravenous substitution with the aim to reach the organs with the blood-flow. There is no doubt that the therapeutic efficacy is ideal for many organs, such as liver, spleen, lung, and skin. All these organs have a good blood circulation and some ability to regenerate.

However, after years of treatment with the already available enzymes, it is shown that some organs are poorly supplied with blood and renewal cycles are slow, the ability to regenerate is decreased. Organs such as bones, cartilage, muscles, cornea, heart valves, meninges or the brain do not show the hope-for effect. All MPS types with brain involvement (neuronopathic forms of MPS types I, II and VII) or predominant skeletal dysplasia (MPS types IVA and B) cannot benefit from enzyme-replacement therapy and do not show the desired improvement.

In animal studies, modifications of glycosylation can change the ability to pass into organs not yet sufficiently reached such as cartilage or bones, but tissue-specific features cannot be sufficiently considered in any artificial production of the enzymes.

Avascular cartilage, heart valves and corneas cannot be reached by blood flow. Also, between blood vessels and brain tissue, several specialised cells form the blood-brain-barrier (BBB) to protect the brain from any unwanted substances in the blood. Therefore, new strategies are necessary to improve the therapeutic efficiency and to provide better outcomes for the affected patients. If patients with MPS I are diagnosed at a very young age, the best option is to treat them with haematopoietic stem cell transplantation (HSCT). Migrating stem cells can reach the brain and other organs, and then differentiate into organ-specific cells producing the missing lysosomal enzymes.

A straightforward method to overcome BBB is the direct injection of a recombinant enzyme into the cerebral fluid. This can be by lumbar puncture (intra-thecal) or intra-ventricular injections in the brain ventricles. Effects can be observed, however unfortunately not all challenges can currently be solved. The liquor flow can be reduced by thickened meninges with storage and vertebral deformities, which are typical for the disease. However, the barrier between cerebral fluid and brain tissue has still not been fully studied. The half-life of enzymes is limited, and the procedure has to be repeated regularly. The clinical trials for patients with MPS I, II, IIIA and IIIB could show some reduced or reversed progression of CNS pathology but long-term effects remain unclear.

Another possibility to overcome BBB is to fuse the enzyme proteins with macromolecules which enter the brain through receptor mediated active transport systems. This physiological transport is known for hormones, neurotransmitters and many other proteins (such as transferrin and insulin). They are transported through the BBB directly into the brain via specific receptores, so, the strategy is to fuse the natural proteins with the artificial enzymes needed in the MPS patient. It is important to note that clinical trials could potentially still show some improvement in affected MPS patients.

Another method is to conjugate the therapeutic enzymes with nano-capsules and to then ferry them across BBB via transcytosis or other transport mechanisms directly into brain cells. Pharmacological chaperones have been proven to be effective in other lysosomal storage diseases such as Gaucher or Fabry disease. Chaperones are able to stabilise three-dimensional conformation of misfolded proteins, such as enzymes. This would be the case of genetic variants causing missense mutation and exchange of only one amino acid in the protein chain. The misfolding pathology reduces stability, half-life and effect of the genetically conditioned enzyme, whereas the chaperone can reverse this disadvantage and increase the activity and efficacy of the enzyme. As a result, pharmacological chaperones are a good option for some diseases and could therefore be an option for some MPS patients in the future.

Some genetic variants cause stop-codons and the production of truncated dysfunctional peptides without any enzymatic activity and degradation within the cell. Stop-codon read through therapy aims for the genetic correction on an RNA level, resulting in the production of a sufficiently functioning gene product. It is already used for some specific mutation for patients with Duchenne muscular dystrophy, but it is too early to predict positive results for patients with MPS I.

Another possibility in the future might be the use of GAG-reducing small molecules such as Genistein, Pentosam polysulfate or Rhodamine B. They are able to influence and/or reduce the synthesis of GAGs which cannot be degraded sufficiently by the genetically changed enzymes with reduced function.

To reduce the GAGs as substrate, could be a chance to create a better relation between substrate and the impaired substrate reducing enzyme. As a result, lysosomal storage could therefore be reduced. Substrate reduction therapy is an established therapeutic concept in some of the other lysosomal storage diseases, but the usefulness in MPS disorders still needs to be proven.

The genetic corrections of DNA sequences in patient cells are no longer only future options as they have now become a reality. Gene variants causing missing or impaired functioning gene products could be replaced by correct genetic sequences and genes. This can be made as an ex vivo approach, where stem cells or fibroblast are removed from the patient and are then cultured in vitro, genetically corrected and consecutively re-injected into the patient.

The genetically corrected DNA in the re-transplanted autologous cells is able to produce correct gene products (in terms of MPS, this is the specific enzyme). The amounts of newly produced enzymes might be sufficient to positively influence the disease course of the treated patients.

An in vivo approach utilises viral vectors which invade cells, and even cell nuclei. Such viruses used are adeno-associated-viruses or lenti-viruses. Such manipulated viruses with the corrective genetic material are directly injected into the patient where they are internalised into deficient cells and are then able to produce the missing gene product. In the case of MPS, the aim is to produce enzyme proteins with sufficient concentrations and activity to prevent the storage of GAGs. Furthermore, clinical trials are underway for several MPS types and therefore, might offer a therapeutic opportunity in early life for affected patients. However, larger studies and a longer follow-up is still needed.

To conclude, MPS are rare genetic disorders and for a long time, they were linked with the myth of being untreatable diseases. Although some of the new therapeutic options are still in clinical trials and not routinely used, the present shows that many of the patients can benefit from the yet available options of HSCT and enzyme replacement therapies. These therapies have an undoubted effect for some of the MPS patients, especially if any form of therapy is started early or if the course of the disease does not affect the nervous system.

However, in the future, new therapeutic options will hopefully bring benefits to those that are not sufficiently improved; the decision of the best therapy will be made on the basis of factors such as the genetic defect, the type of MPS, and the age during treatment. This individualised and personalised therapy will improve the success of MPSs therapies.

Susanne Gerit KircherMedical University of Vienna, AustriaCenter of Pathobiochemistry and Geneticssusanne.kircher@meduniwien.ac.atwww.mps-austria.at

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Mucopolysaccharidoses: future therapies and perspectives - SciTech Europa

Skin Stem Cells Comments Off on Mucopolysaccharidoses: future therapies and perspectives – SciTech Europa | November 29th, 2019

Although one in nine men will receive a prostate cancer diagnosis in his lifetime, cutting-edge research has allowed more men to live longer or even be cured.

One such program that sheds light on this cause is City of Hopes NoShaver November. The month-long fundraising and awareness campaign urges participants to forego shaving to start a conversation, encourage testing and raise critical funds to continue leading-edge prostate cancer research and promising new therapies conducted at City of Hope.

CURE recently spoke with Dr.Tanya Dorff, a medical oncologist at City of Hope who specializes in prostate cancer, about the campaign, her current work and where she sees the future of prostate cancer treatment shifting in the coming years.

CURE: What led you to City of Hope? What do you do there?Dorff: City of Hope was attractive to me because I am a clinical and translational researcher. The reason I came here was to work with scientists who share what we are seeing in the clinic and who incorporate the latest insights from the scientific discoveries in our laboratories into patient care. There is a real sense of mission and urgency that binds scientists to clinicians at City of Hope in a way that is unique and gratifyingly productive.First and foremost, I take care of patients who remain my central inspiration and raison detre, but I spend part of my time writing and running clinical trials that have real potential to impact how we treat patients in the future how we can do even better in the future than we do today. I lead the genitourinary cancer program, which includes fostering collaborations between the incredible physicians from urology, radiation oncology, radiology and pathology to work together both clinically and in research projects.You are Grammy-winning songwriter and vocal producer Kuk Harrells physician. Can you tell me what it was like to treat him?Kuk is such an incredible gentleman; it has been a pleasure to be part of his care team. His attitude toward treatment was one of diligence, and he has approached his illness as an opportunity for personal growth and for giving back by promoting prostate cancer awareness through his story. It has been inspiring to see him come through what was a lengthy and involved treatment with so much positive energy.How has the field of prostate cancer treatment evolved in recent years?More and more men with prostate cancer can be cured, and the men who cannot be cured with todays treatments are clearly living longer and better. This is thanks to new drug approvals in advanced, resistant prostate cancer but even more so to the application of more intensive therapy earlier in the course of the disease. This has been the biggest paradigm shift in prostate cancer over the last five years: up-front intensification in metastatic hormone sensitive prostate cancer.The next big shift in prostate cancer treatment is just now upon us molecular selection of therapies to individualize prostate cancer treatment. The most imminent example is olaparib (Lynparza), a PARP inhibitor, which worked better than standard treatment in patients with castration-resistant prostate cancer whose tumors harbor mutations in DNA repair genes. But the ingenious theranostic approach will be close behind where imaging (scans) show us whether a cancer is expressing a certain target (i.e. PSMA) and if so, a radioactive particle linked to that target is applied (i.e. Lu-177 PMSA).

What are you most hopeful for in cancer treatment in the future?I believe immunotherapy will be the way to durable remission or a cure. Here at City of Hope, we are working hard to improve the effectiveness of immunotherapy for patients with metastatic prostate cancer, studying intensive treatments such as CAR-T and bispecific T-cell engaging antibodies, among other approaches. Our scientists are looking at our patients in real time to learn why treatments work or dont work, and how to better engage the immune system. I am very hopeful that these biologic insights will eventually translate into therapeutic success such as we have seen in leukemia with CAR-T and melanoma with immune checkpoint inhibitors.What advice would you offer someone who has just received a cancer diagnosis of their own?One: Play an active role. Ask questions, and if something doesnt sound right or make sense, ask again. It is so important that patients buy into their treatment, understand and feel confident about the treatment plan. No one is perfect, not even the best doctor, and working together as a team will lead to the best success.

Two: Be a squeaky wheel. Patients who communicate symptoms in real time fare better because problems are addressed before they become more serious.

Three: Stay active. Exercise is one of the things that has been shown over and over again to help cancer survivors and cancer patients in various stages. Obviously, a conversation should occur with the treatment physicians to ensure that there are no restrictions but patients who are more active will come through treatment in better shape.

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Exploring the Future of Prostate Cancer with City of Hope - Curetoday.com

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If are fasting intermittently or taking long food breaks, here's the news for you. A new study presented at the 2019 American Heart Association Scientific Sessions in Philadelphia has found the good health outcomes of intermittent fasting for cardiac catheterisation patients.The study showed that patients who practised intermittent fasting lived longer than those who didn't. In addition, they are less likely to be diagnosed with heart failure. Cardiac catheterisation is a procedure used to diagnose and treat certain cardiovascular conditions. "It is another example of how we're finding that regular fasting can lead to better health outcomes and longer lives," said the study's principal investigator Benjamin Horne, PhD, director of cardiovascular and genetic epidemiology at the Intermountain Healthcare Heart Institute.Researchers asked 2,001 intermountain patients undergoing cardiac catheterization from 2013 to 2015 a series of lifestyle questions, including whether or not they practised routine intermittent fasting. They then followed up with those patients 4.5 years later and found that routine intermittent fasters had a greater survival rate than those who did not.Because people who fast routinely also are known to engage in other healthy behaviours, the study also evaluated other parameters including demographics, socioeconomic factors, cardiac risk factors, comorbid diagnoses, medications and treatments, and other lifestyle behaviours like smoking and alcohol consumption.Correcting statistically for these factors, long-term routine fasting remained a strong predictor of better survival and lower risk of heart failure, according to researchers. While the study does not show that fasting is the causal effect for better survival, these real-world outcomes in a large population do suggest that fasting may be having an effect and urge continued study of the behaviour. "While many rapid weight loss fasting diets exist today, the different purposes of fasting in those diets and in this study should not be confused with the act of fasting," said Dr Horne. "All proposed biological mechanisms of health benefits from fasting arise from effects that occur during the fasting period or are consequences of fasting," he added.Why long-term intermittent fasting leads to better health outcomes is still largely unknown, though Dr Horne said it could be a host of factors. Fasting affects a person's levels of haemoglobin, red blood cell count, human growth hormone, and lowers sodium and bicarbonate levels, while also activating ketosis and autophagy - all factors that lead to better heart health and specifically reduce risk of heart failure and coronary heart disease. "With the lower heart failure risk that we found, which is consistent with prior mechanistic studies, this study suggests that routine fasting at a low frequency over two-thirds of the lifespan is activating the same biological mechanisms that fasting diets are proposed to rapidly activate," Dr Horne noted.Researchers speculate that fasting routinely over a period of years and even decades conditions the body to activate the beneficial mechanisms of fasting after a shorter length of time than usual.

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Can intermittent fasting help you live long? - Times of India

Cardiac Stem Cells Comments Off on Can intermittent fasting help you live long? – Times of India | November 29th, 2019

VANCOUVER, British Columbia, Nov. 26, 2019 (GLOBE NEWSWIRE) -- Novoheart (Novoheart or the Company) (TSXV: NVH; FWB: 3NH), a global stem cell biotechnology company, is pleased to announce a collaboration with global biopharmaceutical company AstraZeneca, in an effort to develop the worlds first human-specific in vitro, functional model of heart failure with preserved ejection fraction (HFpEF), a common condition especially among the elderly and in women, with the reported prevalence approaching 10% in women over the age of 80 years.1

Heart failure (HF) is a global pandemic with an estimated 64.3 million cases worldwide in 2017, with an increasing trend in prevalence2. The annual global economic burden of HF is estimated at over US$100 billion3. Accounting for approximately 50% of HF cases, HFpEF in particular is a major and growing public health problem worldwide, with its pathological mechanisms and diverse etiology poorly understood. Due to these complexities, models of the disease available to date, including various animal models, have limited ability to mimic the clinical presentation of HFpEF4. Therefore, drug developers lack an effective tool for preclinical testing of drug candidates for efficacy, and as a result, clinical outcomes for HFpEF have not improved over the last decades, with no effective therapies available.

In collaboration with the Cardiovascular, Renal and Metabolism therapy area of AstraZeneca, the initial phase of the project aims to establish a new in vitro model, leveraging Novohearts proprietary 3-D human ventricular cardiac organoid chamber (hvCOC) technology, that reproduces key phenotypic characteristics of HFpEF. Also known as human heart-in-a-jar, the hvCOC is the only human engineered heart tissue available on the market to date that enables clinically informative assessment of human cardiac pump performance including ejection fraction and developed pressure. Unlike animal models, engineered hvCOCs can be fabricated with specific cellular and matrix compositions, and patient-specific human induced pluripotent stem cells (iPSCs), that allow control over their physical and mechanical properties to mimic those observed in HFpEF patient hearts. Together with Novohearts proprietary hardware and software, this aims to provide a unique assay for understanding the mechanisms of HFpEF, identification of new therapeutic targets, and assessment of novel therapeutics for treating HFpEF patients. Novoheart will exclusively own the intellectual property rights to the newly developed HFpEF hvCOC model.

We are delighted to partner with AstraZeneca, an organization which has long invested in cardiovascular research and is committed to bringing new therapeutic solutions to patients with heart failure, said Novoheart CSO, Dr. Kevin Costa. We look forward to co-developing this new HFpEF hvCOC model into a powerful new tool in the worldwide battle against heart failure.

Regina Fritsche Danielson, Senior Vice President, Head of Research and Early Development, Cardiovascular, Renal and Metabolism, BioPharmaceuticals R&D, AstraZeneca, said, There are significant unmet treatment needs in patients with heart failure with preserved ejection fraction. By combining Novohearts proprietary hvCOC model with our expertise in heart failure, we aim to create the first in vitro model reproducing phenotypic characteristics of heart failure with preserved ejection fraction. This could bridge the gap between in vivo animal models and clinical trials to help accelerate the drug discovery process by providing human-specific preclinical data.

1 Heart Fail Clin. 2014; 10(3):377388.2 Lancet. 2018; 392:1789-1858.3 Int J Cardiol. 2014; 171(3):368-76.4 JACC Basic Transl Sci. 2017; 2(6):770-789.

About Novoheart:

Novoheart is a global stem cell biotechnology company pioneering an array of next-generation human heart tissue prototypes. It is the first company in the world to have engineered miniature living human heart pumps that can revolutionize drug discovery, helping to save time and money for developing new therapeutics. Also known as 'human heart-in-a-jar', Novohearts bio-artificial human heart constructs are created using state-of-the-art and proprietary stem cell and bioengineering approaches and are utilized by drug developers for accurate preclinical testing of the effectiveness and safety of new drugs, maximizing the successes in drug discovery whilst minimizing costs and harm caused to patients. With the recent acquisition of Xellera Therapeutics Limited for manufacturing Good Manufacturing Product (GMP)-grade clinical materials, Novoheart is now developing gene- and cell-based therapies as well as next-generation therapeutics for cardiac repair or regeneration.

For further information, please contact:Ronald Li, CEOinfo@novoheart.com

For media enquiries or interviews, please contact:Media Relationsmedia@novoheart.com

Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

Cautionary Note Regarding Forward-Looking Statements

Information set forth in this news release may involve forward-looking statements under applicable securities laws. Forward-looking statements are statements that relate to future, not past, events. In this context, forward-looking statements often address expected future business and financial performance, and often contain words such as "anticipate", "believe", "plan", "estimate", "expect", and "intend", statements that an action or event "may", "might", "could", "should", or "will" be taken or occur, or other similar expressions. By their nature, forward-looking statements involve known and unknown risks, uncertainties and other factors which may cause the actual results, performance or achievements, or other future events, to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Such factors include, among others, the risks identified in under the heading Risk Factors in Novohearts annual information form for the year ended June 30, 2019 or other reports and filings with the TSX Venture Exchange and applicable Canadian securities regulators. Forward-looking statements are made based on management's beliefs, estimates and opinions on the date that statements are made and the respective companies undertakes no obligation to update forward-looking statements if these beliefs, estimates and opinions or other circumstances should change, except as required by applicable securities laws. Investors are cautioned against attributing undue certainty to forward-looking statements.

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Novoheart to Co-develop First of its Kind Human Heart-in-a-Jar Model of Heart Failure with AstraZeneca - GlobeNewswire

Cardiac Stem Cells Comments Off on Novoheart to Co-develop First of its Kind Human Heart-in-a-Jar Model of Heart Failure with AstraZeneca – GlobeNewswire | November 29th, 2019

Hailie and Treylin Hyman saw the bruising on their baby girls leg as a symbol that the active 1-year-old was getting to walk.

But as a blood test would following disclose and reveal, little Maci was suffering from an extremely unusual blood cancer that scared her life outwardly a risky treatment a practice nearly as serious as the disease.

At the start, it was very scary, Hailie Hyman told the reports.

Terrifying periods followed the diagnosis, punctuated by one crucial difficulty after another, starting the Boiling Springs couple to wonder if Maci would remain and survive or not.

The Hymans course started last February at Macis 1-year-old well-child checkup.

We had no clue anything was incorrect, her mom told. But they did a normal (blood test) and a few hours later, we attended a call telling her platelets were very low.

The Hymans was transferred to a hematologist who gained other abnormalities in Macis blood and listed a bone marrow biopsy to examine further.

During the treatment, the child endured an aneurysm in an artery and progressed into cardiac arrest. The medical team gave CPR for 20 minutes before she was steadied, her mom told.

Later, in the Emergency room, she underwent internal bleeding, too.

It was difficult, she told. There were many times that I would just pray and pray and pray.

Initially considering Maci had leukemia, doctors finally discovered she had myelodysplastic syndrome or MDS.

The situation occurs when abnormal cells in the bone marrow leave the patient weak and unable to make adequate blood.

In children, its more uncommon still. Most people are diagnosed in their 70s.

Maci had to produce regular blood transfusions, antibiotics, and other medicines to struggle the MDS, Bryant stated. But the only support for a remedy was a stem cell transplant.

The transplant is very risky.

Its also laden with possibly life-threatening difficulties, including graft vs. host disease, which happens when immune cells from the donor strike the patients body, Bryant told. Other difficulties incorporate permanent kidney damage and gastrointestinal problems.

There were so many moments during her initial months that it appeared like she would not survive, Bryant stated. So the fact that she is here is a miracle.

Macis family got an anonymous donor by the National Marrow Donor Program, participating many individuals to register in the process, Bryant told.

Maci was admitted to MUSC on June 5 and discharged on Oct. 14.

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Toddler Bravely Cheats Death After He Survived Rare Cancer And Its Treatment - The Digital Weekly

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ANI | Updated: Nov 29, 2019 20:58 IST

Washington D.C. [USA], Nov 29 (ANI): Researchers have discovered a stem cell therapy that might help the heart recuperate from an attack.This study published in the journal Nature reported that injecting living or even dead heart stem cells into the injured hearts of mice triggers an acute inflammatory process, which in turn generates a wound healing-like response to enhance the mechanical properties of the injured area.Mediated by macrophage cells of the immune system, the secondary healing process provided a modest benefit to heart function after a heart attack, according to the principal investigator Jeffery Molkentin, PhD, director of Molecular Cardiovascular Microbiology a Cincinnati Children's Hospital Medical Center and a professor of the Howard Hughes Medical Institute (HHMI)."The innate immune response acutely altered cellular activity around the injured area of the heart so that it healed with a more optimized scar and improved contractile properties," Molkentin said.The findings build on a 2014 study published by the same research team. As in that earlier study, the current paper shows that injecting c-kit positive heart stem cells into damaged hearts as a strategy to regenerate cardiomyocytes doesn't work.The findings prompted Molkentin and his colleagues to conclude that there is a need to "re-evaluate the current planned cell therapy based clinical trials to ask how this therapy might really work."Researchers worked with two types of heart stem cells currently used in the clinical trials -- bone marrow mononuclear cells and cardiac progenitor cells.As they went through the process of testing and re-verifying their data under different conditions, they were surprised to discover that in addition to the two types of stem cells, injecting dead cells or even an inert chemical called zymosan also provided benefit to the heart by optimizing the healing process. Zymosan is a substance designed to induce an innate immune response.They reported that stem cells or zymosan therapies tested in this study altered immune cell responses that significantly decreased the formation of extracellular matrix connective tissue in the injury areas, while also improving the mechanical properties of the scar itself.Researchers also found that stem cells and other therapeutic substances like zymosan have to be injected directly into the hearts surrounding the area of infarction injury."Most of the current trials were also incorrectly designed because they infuse cells into the vasculature. Our results show that the injected material has to go directly into the heart tissue flanking the infarct region. This is where the healing is occurring and where the macrophages can work their magic," Molkentin explained. (ANI)

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Researchers discover a stem cell therapy that can help heal injured heart - ANI News

Bone Marrow Stem Cells Comments Off on Researchers discover a stem cell therapy that can help heal injured heart – ANI News | November 29th, 2019

A TODDLER born with two types of leukaemia will spend Christmas at home after recovering from a second stem cell transplant.

Amelia Topa, who turned two on Wednesday, was donated cells harvested from a newborn babys umbilical cord blood flown in from the US.

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The brave youngster is now in remission and is preparing to enjoy the festive season with relieved parents Kerri Paton, 23, and Igor Topa, 24, in Turriff, Aberdeenshire.

Recalling Junes life-saving op, mum Kerri said: Amelia soared through the transplant and shes doing really well. I couldnt be prouder.

Its rare enough to be born with leukaemia but to be born with a mix of two kinds is almost unheard of.

Worried medics alerted Amelias parents to raised purple spots on her body shortly after she was born at Dr Grays Hospital, Elgin.

She was diagnosed with acute lymphoblastic leukaemia and acute myeloid leukaemia and spent Christmas 2017 in hospital.

The tot was given a bone marrow transplant six months later using stem cells donated by a man aged between 16 and 30.

The treatment worked but tests showed Amelias cancer had returned in February.

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She endured gruelling chemo before her second transplant in the summer.

Kerri added: I hope Amelias story will help other families going through cancer. There is a light at the end of the tunnel.

Amelia has now been chosen to receive Cancer Research UKs first Children & Young People Star Award.

The prize, backed by TK Maxx and stars including Dame Emma Thompson, is open to under-18s battling cancer or who have been treated in the last five years.

The charitys Lisa Adams said: Were calling on families to nominate inspirational youngsters so we can recognise their courage.

VISIT cruk.org/childrenand youngpeople to nominate.

scottish-sun@the-sun.co.uk

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Brave Scots tot born with two types of leukaemia to spend Christmas at home after second stem cell transplant - The Scottish Sun

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Hematopoietic stem cells (HSCs) can be used to better understand and treat blood-based diseases. Stem cell research in the model organism zebrafish is well-studied in the developmental stage, but is limited in the adult stage because HSCs are difficult to purify in this species. Researchers at Kanazawa University and their collaborators have developed a new purification scheme that allows HSCs to be purified from adult zebrafish kidneys, potentially opening new possibilities for stem cell research.

Kanazawa, Japan Hematopoietic stem cells (HSCs) are multipotent cells that can develop into every type of blood cell in the body. They can also be used in medical research to understand and treat blood-based diseases. Zebrafish (Danio rerio) are used to study HSCs, particularly in the field of developmental biology, but the research in the adult animal is often limited because stem cells are difficult to purify in this species. Researchers at Kanazawa University and their collaborators now describe a purification scheme that allows these elusive zebrafish HSCs to be collected.

Zebrafish are a great system to study how hematopoietic cells function in normal development and their role in disease, says lead researcher Isao Kobayashi. Much of their biology mirrors what we see in humans, and with zebrafish theres the added benefit of having quite a few experimental tools at our fingertips, including live cell imaging and comparative analysis among vertebrates. Unfortunately, its proven challenging to effectively isolate HSCs from this species, and this has been a major impediment to the field.

HSCs are highly abundant in the kidneys of adult zebrafish (unlike in humans, where HSCs are found in bone marrow). The challenge is separating them from other cells found in kidneys. Cell separation usually involves a purification technique called flow cytometry, where cells are sent in single file through a tube and hit with a laser beam. The machine (a flow cytometer) then sorts the cells based on how they reflect or scatter light.

In the study, published in Scientific Reports, the researchers created a strain of zebrafish that makes two light-emitting proteins, one green (Green Fluorescent Protein, GFP) and one red (mCherry), that can be sensed and sorted by a flow cytometer. Each fluorescent protein in this zebrafish strain was regulated by the genes related with blood cells, but the cells having both fluorescent proteins were limited in HSCs. By color coding the cells with two distinct blood cell markers, the team was able to purify cells that show hallmark signs stemness like the ability to self-propagate and differentiate into other types of blood cells.

So, what might the successful isolation of HSCs in zebrafish mean for the field of stem cell research?

When HSCs were finally purified in mice, the research community learned an enormous amount about how and where stem cells self-renew and differentiate to form blood cells, says co-author Mao Kondo. Were very hopeful that this might spur a similar proliferation of research in zebrafish. In addition to some experimental advantages in zebrafish, we found that zebrafish HSCs share many key genes in common with HSCs in mammals. This suggests that mechanistic discoveries in zebrafish could have direct implications for understanding blood diseases in humans and for developing new medical treatments.

Figure.

Hematopoietic stem cells can be isolated as gata2a:GFP+ runx1:mCherry+ (gata2a+ runx1+) cells in the zebrafish kidney by flow cytometry (left panels). Transplantation assays confirmed the hematopoietic potential of gata2a+ runx1+cells (right panels).

Article

Enrichment of hematopoietic stem/progenitor cells in the zebrafish kidney

Journal: Scientific Reports

Authors: Isao Kobayashi, Mao Kondo, Shiori Yamamori, Jingjing Kobayashi-Sun, Makoto Taniguchi, Kaori Kanemaru, Fumihiko Katakura & David Traver

DOI: 10.1038/s41598-019-50672-5

Funder

This work was supported in part by Grant-in-Aid for Young Scientists (B) from the Japan Society for the Promotion of Science (17K15393).

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Researchers Find a Way to Collect Elusive Blood Stem Cells from Zebrafish - Mirage News

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This Mesenchymal Stem Cells research study consists of the historical data from and forecasts till 2026. The report is created keeping in mind to make it a valuable source of information for market specialists in readily accessible documents. The documents are curated with clearly presented graphs and figures.

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The global mesenchymal stem cells market size to reach USD 2,518.5 Million by 2026, growing at a CAGR of 7.0% during forecast period, according to a new research report published by Alexa Reports Research. The report Mesenchymal Stem Cells Market, [By Source (Bone Marrow, Umbilical Cord Blood, Peripheral Blood, Lung Tissue, Synovial Tissues, Amniotic Fluids, Adipose Tissues); By Application (Injuries, Drug Discovery, Cardiovascular Infraction, Others); By Region]: Market Size & Forecast, 2018 2026 provides an extensive analysis of present market dynamics and predicted future trends. The market was valued at USD 1,335.1 million in 2017. In 2017, the drug discovery application dominated the market, in terms of revenue. North America region is observed to be the leading contributor in the global market revenue in 2017.

are adult stem cells, which are traditionally found in the bone marrow. However, they can also be parted from other available tissues including peripheral blood, cord blood, fallopian tube. These stem cells mainly function for the replacement of damaged cell and tissues. The potential of these cell is to heal the damaged tissue with no pain to the individual. Scientists are majorly focusing on developing new and innovative treatment options for the various chronic diseases like cancer. Additionally, the local governments have also taken various steps for promoting the use of these stem cells.

The significant aspects that are increasing the development in market for mesenchymal stem cells consist of enhancing need for these stem cells as an efficient therapy option for knee replacement. Raising senior populace throughout the world, as well as increasing frequency of numerous persistent conditions consisting of cancer cells, autoimmune illness, bone and cartilage diseases are elements anticipated to enhance the market development throughout the forecast period. The mesenchymal stem cells market is obtaining favorable assistance by the reliable federal government policies, as well as funding for R&D activities which is anticipated to influence the market growth over coming years. According to the reports released by world health organization (WHO), by 2050 individuals aged over 60 will certainly make up greater than 20% of the globes population. Of that 20%, a traditional quote of 15% is estimated to have symptomatic OA, as well as one-third of these individuals are expected to be influenced by extreme specials needs. Taking into consideration all these aspects, the market for mesenchymal stem cells will certainly witness a substantial development in the future.

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Increasing demand for better healthcare facilities, rising geriatric population across the globe, and continuous research and development activities in this area by the key players is expected to have a positive impact on the growth of Mesenchymal Stem Cells market. North America generated the highest revenue in 2017, and is expected to be the leading region globally during the forecast period. The Asia Pacific market is also expected to witness significant market growth in coming years. Developing healthcare infrastructure among countries such as China, India in this region is observed to be the major factor promoting the growth of this market during the forecast period.

The major key players operating in the industry are Cell Applications, Inc., Cyagen Biosciences Inc. Axol Bioscience Ltd., Cytori Therapeutics Inc., Stem cell technologies Inc., Celprogen, Inc. BrainStorm Cell Therapeutics, Stemedica Cell Technologies, Inc. These companies launch new products and undertake strategic collaboration and partnerships with other companies in this market to expand presence and to meet the increasing needs and requirements of consumers.

Alexa Reports Research has segmented the global mesenchymal stem cells market on the basis of source type, application and region:

Mesenchymal Stem Cells Source Type Outlook (Revenue, USD Million, 2015 2026)

Bone MarrowUmbilical Cord BloodPeripheral BloodLung TissueSynovial TissuesAmniotic FluidsAdipose Tissues

Mesenchymal Stem Cells Application Outlook (Revenue, USD Million, 2015 2026)

InjuriesDrug DiscoveryCardiovascular InfractionOthers

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About Us:Alexa Reports is a globally celebrated premium market research service provider, with a strong legacy of empowering business with years of experience. We help our clients by implementing decision support system through progressive statistical surveying, in-depth market analysis, and reliable forecast data. Alexa Reports is a globally celebrated premium market research service provider, with a strong legacy of empowering business with years of experience. We help our clients by implementing decision support system through progressive statistical surveying, in-depth market analysis, and reliable forecast data.

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Systematic analysis and evaluation of Mesenchymal Stem Cells Market with share, growth rate, Forecasts to 2026 - WindStreetz

Bone Marrow Stem Cells Comments Off on Systematic analysis and evaluation of Mesenchymal Stem Cells Market with share, growth rate, Forecasts to 2026 – WindStreetz | November 29th, 2019

GammaDelta Therapeutics is a company that focusses on utilizing the unique properties of gamma delta () T cells to develop novel immunotherapies for patients.Through their research, the companys scientists have discovered a number of targets and antibodies that have the potential to modulate the activity of T-cells in situ. Therefore, GammaDelta Therapeutics recently announced the formation of Adaptate Biotherapeutics, a spin-out company that will focus on research in this area.

Technology Networks spoke with Natalie Mount, CEO of Adaptate BioTherapeutics, to learn more about the company's aims and the challenges faced when developing immunotherapies and advancing them into clinical studies.

Molly Campbell (MC) Please can you tell us more about T-cell based cell therapy products and their potential applications?Natalie Mount (NM): T cells play an increasingly appreciated critical role in immune surveillance, being able to recognize malignant/transformed cells through a pattern of stress markers. The recognition mechanism is not major histocompatibility complex (MHC) restricted and not dependent on a single antigen.

T cells therefore have potential in a range of disease indications, including both hematological and solid malignancies and a positive correlation between T cell infiltration and prognosis/survival in patients has been determined in a range of oncology indications in studies published in the literature by other groups. Additionally, as a cell therapy, T cells can be used in an allogeneic setting (ie, T cells can be used for unrelated recipients without a requirement for matching).

Both Adaptate Biotherapeutics and GammaDelta Therapeutics are focussed on harnessing the potential of T cells, in particular the V1 subtype which is the predominant T cell type in tissue.This is based on data originating from the labs of Professor Adrian Hayday of Kings College London and the Crick Institute, supported by Cancer Research Technology and also from Professor Bruno Silva Santos of Institute for Molecular Medicine at the University of Lisbon, Portugal.

Previous clinical trials conducted by other groups/companies targeting or using T cells in cancer have focussed on the V2 subtype which is predominant in the blood. These trials have demonstrated safety, but efficacy has been limited.Compared to V2 cells, V1 cells, which are the focus of work at Adaptate Biotherapeutics and GammaDelta Therapeutics, are less susceptible to exhaustion and activation induced cell death. Expansion of donor derived V1 has been shown to be a positive prognostic indicator for acute myeloid leukemia patients following hematopoietic stem cell transplant.

MC: Why are current immunotherapy treatment approaches limited?NM: Immunotherapy approaches have had very significant success and impact in Oncology recently, however, challenges and unmet needs remain.One challenge is effective treatment of solid tumors. The hypoxic, low nutrient tumor environment provides a challenge for successful infiltration and activation of T cells. However, V1 T cells have real potential as they are naturally tissue resident and hence primed for this environment. In addition, their ability to recognize malignant cells by a pattern of markers expressed by dysregulated, transformed cells rather than one specific antigen presented by the MHC provides an additional advantage for both specificity of response and maintenance of efficacy.

T cells act as orchestrators of an immune response and, following recognition of a cell as malignant, they induce maturation of monocytes and signal to alpha beta T cells, hence increasing immunogenicity of the tumor and providing a sustained response, with potential even in tumors with low mutational load which have proven challenging with other immunotherapies.

MC: The new spin-out company, Adaptate Biotherapeutics, will build on GammaDelta's knowledge to modulate T-cell activity using therapeutic antibodies. Why have you decided to create a spin-out focusing on this area of research?NM: GammaDelta Therapeutics was formed in 2016 to harness the unique properties of T cells, and since then has gained extensive knowledge of T-cell biology. In addition to gaining insight into cell growth and isolation, the companys scientists have also discovered a number of targets and antibodies that have potential to modulate the activity of T-cells in situ.

GammaDelta Therapeutics now has a pipeline of cell therapy products progressing into clinical development under the guidance of CEO, Dr Paolo Paoletti.

Adaptate Biotherapeutics will be developing antibodies which will be administered to cancer patients to modulate activity of the patient's gamma delta T cells in situ.

Delivery of cell therapy and antibody therapeutics each needs focus and specific skillsets and formation of two independent entities will facilitate this. The two companies share a common goal to harness the potential of T cells to bring effective therapies to patients. Both benefit from support of the scientific founding team and have common investors, Abingworth and Takeda Pharmaceuticals.MC; Your goal is to develop targets and antibodies that can modulate the activity of T-cells and advance them into clinical studies. What challenges exist here, and how do you hope to overcome them?

Our assets at Adaptate Biotherapeutics are currently at the pre-clinical stage and therefore face the non-clinical development risks for a novel therapy. However, these risks are mitigated by biology understanding from our scientific founders and the work at GammaDelta Therapeutics to date.

One of our challenges is in selecting the most suitable patient population for initial trials. There is potential for opportunity for our therapeutics in multiple indications but the utility of animal models in modelling the human immune compartment and human tumor setting is limited. Therefore in vitro and ex vivo models are important, in addition to the learnings from other clinical studies.

MC: GammaDelta Therapeutics formed in 2016 to gain extensive knowledge of T-cell biology and to developing a portfolio of investigational cell therapies. Some of these cell therapies are poised to enter clinical development. Can you tell us any further information about these therapies?NM: GammaDelta was set up to develop cell-based therapy utilizing ex-vivo expanded tissue resident gd T cells. Subsequent acquisition of Lymphact SAS allowed GammaDelta to augment its capabilities with a platform for ex-vivo expansion of blood derived V1 cells. GammaDelta is focussed on progressing ex-vivo expanded skin and blood derived V1 cells to the clinic both in unengineered and engineered formats. Clinical trials are currently on track to commence in the next 12-18 months.

MC: Your press release states: "The two companies will continue sharing their insights into T-cell biology as they work towards developing different therapeutic modalities". How will you continue to share insights here?NM: Antibodies and cells represent complementary approaches to realizing the potential of T cell activity for patients with solid and haematological malignancies.

The two companies will work together in areas of common interest in the biology of these fascinating cells, such as understanding the phenotype and behavior of T cells in tumors and mechanisms of cell regulation as well as the effects of antibody on the T cells.

We have deliberately established a contractual framework that allows efficient collaboration between scientists of both the companies via formal and informal meetings.

MC: What are your hopes for the future of Adaptate Biotherapeutics?NM: This is a remarkable time in the development of new immune therapies, and the role of "non-conventional" cell types of the immune system is coming to the fore as we recognize the successes achieved to date and the needs of patients and related scientific challenges that remain.

Both GammaDelta Therapeutics and Adaptate Biotherapeutics are at the lead of translating our increasing understanding of T cell biology and its potential into therapies to address these unmet needs.

Adaptate Biotherapeutics has a fantastic opportunity to build and accelerate a portfolio of antibody-based approaches in this novel area and I look forward to the successful translation of this science into therapies with the support of our investors at Abingworth and Takeda Pharmaceuticals.

Dr Natalie Mount, CEO of Adaptate Biotherapeutics was speaking with Molly Campbell, Science Writer, Technology Networks.

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Harnessing Gamma T Cells To Bring Effective Therapies to Patients - Technology Networks

Skin Stem Cells Comments Off on Harnessing Gamma T Cells To Bring Effective Therapies to Patients – Technology Networks | November 28th, 2019

.

The Black Friday sales are almost among us, meaning the halls of your local department store are about to become a battleground. (The smartest of shoppers know the key to maintaining sanity is to do it all online.)

But when faced with deals and discounts from every angle, its crucial to keep your cool. You dont want to panic-buy all those as-seen-on-Instagram products before youre covered the essentials you know, the things you'll use day in, day out, until they're empty.

Resist those shiny, sparkling impulse buys (although yes, that glitter lipstick would look pretty good on NYE), and youll come out triumphant with a well-curated skincare edit that will keep your skin happy long after the last Quality Street has been polished off.

So, skip the scrolling and head straight for the good stuff this year. Here, discover the eight most unequivocally iconic beauty products we've spotted in the Black Friday sales. Trust us: you cannot go wrong with any (or all) of these heroes.

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1Soleil Tan De Chanel

Chanel

WAS 40 NOW 36

Soleil Tan de Chanel is the kind of product beauty dreams are made of. The weighty pot will last you all year, while the lacquered, double-C engraved pot will look incredibly bouji on your dressing table.

But of course,it's what's on the inside that really counts, and boy is this a brilliantly formulated bronzer. Unlike literally anything else on the market (many brands have tried and failed to 'dupe' it), this dense, mattecream can be picked up with a powder brush, dusted under or over foundation, and swiped strategically to carve out killer cheekbones it's revolutionary.

2Blanche Eau de Parfum

Byredo

WAS 110 NOW 93.50

Byredo's perfumes are coveted the world over, but with so many to choose from, you'll be hard-pressed to find a favourite without some kind of divine intervention.

Our advice? Head straight for Blanche, which is a byword for the crispest, cleanest scent you'll ever spritz. Forget cloying talc notes or blink-and-you'll-miss-it citrus: this is pure violet and musk-tinted freshness, like the cleanest cotton sheets that ever were.

And seeing as there's a very generous 15% discount (thank you, Liberty), maybe treat yourself to the body wash, too.

3The Rich Cream

205.00

WAS 205 NOW 174

The hype around Augustinus Bader's debut product was remarkable across the world, A-listers and beauty editors fawned. (Victoria Beckham even tapped him up to create her debut skincare product.)

Turns out, this clever cream really delivers. The secret is the Trigger Factor Complex, which works to kick-start the natural healing process of the body's stem cells. 30 years of research and development have clearly paid off.

4Bronzing Powder

Narsasos.com

WAS 31 NOW 24.50

The name of many a Nars product precedes its performance Orgasm, anyone? But not this one: the brand's Laguna and Casino bronzing powdersare famed for their supreme performance alone.

Both illuminate without relying on glitter (which always looks fake), and are warming without imparting those giveaway ruddy undertones.

Paler skins are destined for Laguna, while darker tones will love Casino. Your bank balance will love either.

5Diorshow Pump 'N' Volume HD Mascara

DIOR

WAS 28 NOW 25.20

Ask any beauty editor what their ride-or-die mascara is and you'll get...a lot of conflicting opinions.

Like the perfect shade of red lipstick, a favourite mascara is a subjective thing. After all,few can deliver perfection when it comes to length, volumeand colour. Enter Dior's Pump 'N' Volume: the mascara to unite us all. (And yes, it's the one with the no-wastesqueezy tube.)

6Do Son Eau De Parfum

Diptyque

WAS 120 NOW 96

Many of Diptyque's fragrances could be considered iconic, but Do Son is the one that'll see you being stopped by strangersin the street.

The tuberose trail is enticing enough, but it's the unusual addition of orange blossom and jasmine that take things to truly memorable heights. A spectacularode to its namesake beach, in Vietnam's Ha Long Bay.

7Ruby Woo Matte Lipstick

WAS 17.50 NOW 14

If you're a 'lipstick person', you already know about this one. You've likely got one stashed in your bag right now, as well as one on your dresser, and an 'emergency' onefloating around your bedroom somewhere.

So good it's never been successfully imitated, MAC's Ruby Woo is the ultimate lipstick. A true red, it's neither too orange or too blue, and there's no skin tone it won't look beautiful against.

8Glow Tonic

WAS 18 NOW 15.30

We all know that alpha-hydroxy-acids are the gold standard when it comes to resurfacing, but so many require a degree in dermatology to use correctly.

Forgo the faffing in favour of Pixis cult Glow Tonic: it might look cute, but its packed with 5% brightening glycolic acid alongside soothing aloe vera. Simply sweep it over cleansed skin nightly no brow-furrowing required.

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The 8 Most Iconic Beauty Products In The Black Friday Sales To Buy Now - elle.com

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DUBLIN, Nov. 28, 2019 /PRNewswire/ -- The "Genome Editing Services Market-Focus on CRISPR 2019-2030" report has been added to ResearchAndMarkets.com's offering.

This report features an extensive study of the current landscape of CRISPR-based genome editing service providers. The study presents an in-depth analysis, highlighting the capabilities of various stakeholders engaged in this domain, across different geographical regions.

Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques. The biopharmaceutical pipeline includes close to 500 gene therapies, several of which are being developed based on the CRISPR technology.

Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated. However, successful gene manipulation efforts involve complex experimental protocols and advanced molecular biology centered infrastructure. Therefore, many biopharmaceutical researchers and developers have demonstrated a preference to outsource such operations to capable contract service providers.

Consequently, the genome editing contract services market was established and has grown to become an indispensable segment of the modern healthcare industry, offering a range of services, such as gRNA design and construction, cell line development (involving gene knockout, gene knockin, tagging and others) and transgenic animal model generation (such as knockout mice). Additionally, there are several players focused on developing advanced technology platforms that are intended to improve/augment existing gene editing tools, especially the CRISPR-based genome editing processes.

Given the rising interest in personalized medicine, a number of strategic investors are presently willing to back genetic engineering focused initiatives. Prevalent trends indicate that the market for CRISPR-based genome editing services is likely to grow at a significant pace in the foreseen future.

Report Scope

One of the key objectives of the report was to evaluate the current opportunity and the future potential of CRISPR-based genome editing services market. We have provided an informed estimate of the likely evolution of the market in the short to mid-term and long term, for the period 2019-2030.

In addition, we have segmented the future opportunity across [A] type of services offered (gRNA construction, cell line engineering and animal model generation), [B] type of cell line used (mammalian, microbial, insect and others) and [C] different geographical regions (North America, Europe, Asia Pacific and rest of the world).

To account for the uncertainties associated with the CRISPR-based genome editing services market and to add robustness to our model, we have provided three forecast scenarios, portraying the conservative, base and optimistic tracks of the market's evolution.

The research, analysis and insights presented in this report are backed by a deep understanding of key insights generated from both secondary and primary research. All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

Key Topics Covered

1. PREFACE1.1. Scope of the Report1.2. Research Methodology1.3. Chapter Outlines

2. EXECUTIVE SUMMARY

3. INTRODUCTION3.1. Context and Background3.2. Overview of Genome Editing3.3. History of Genome Editing3.4. Applications of Genome Editing3.5. Genome Editing Techniques3.5.1. Mutagenesis3.5.2 Conventional Homologous Recombination3.5.3 Single Stranded Oligo DNA Nucleotides Homologous Recombination3.5.4. Homing Endonuclease Systems (Adeno Associated Virus System)3.5.5. Protein-based Nuclease Systems3.5.5.1. Meganucleases3.5.5.2. Zinc Finger Nucleases3.5.5.3. Transcription Activator-like Effector Nucleases3.5.6. DNA Guided Systems3.5.6.1. Peptide Nucleic Acids3.5.6.2. Triplex Forming Oligonucleotides3.5.6.3. Structure Guided Endonucleases3.5.7. RNA Guided Systems3.5.7.1. CRISPR-Cas93.5.7.2. Targetrons3.6. CRISPR-based Genome Editing3.6.1. Role of CRISPR-Cas in Adaptive Immunity in Bacteria3.6.2. Key CRISPR-Cas Systems3.6.3. Components of CRISPR-Cas System3.6.4. Protocol for CRISPR-based Genome Editing3.7. Applications of CRISPR3.7.1. Development of Therapeutic Interventions3.7.2. Augmentation of Artificial Fertilization Techniques3.7.3. Development of Genetically Modified Organisms3.7.4. Production of Biofuels3.7.5. Other Bioengineering Applications3.8. Key Challenges and Future Perspectives

4. CRISPR-BASED GENOME EDITING SERVICE PROVIDERS: CURRENT MARKET LANDSCAPE4.1. Chapter Overview4.2. CRISPR-based Genome Editing Service Providers: Overall Market Landscape4.2.3. Analysis by Type of Service Offering4.2.4. Analysis by Type of gRNA Format4.2.5. Analysis by Type of Endonuclease4.2.6. Analysis by Type of Cas9 Format4.2.7. Analysis by Type of Cell Line Engineering Offering4.2.8. Analysis by Type of Animal Model Generation Offering4.2.9. Analysis by Availability of CRISPR Libraries4.2.10. Analysis by Year of Establishment4.2.11. Analysis by Company Size4.2.12. Analysis by Geographical Location4.2.13. Logo Landscape: Distribution by Company Size and Location of Headquarters

5. COMPANY COMPETITIVENESS ANALYSIS5.1. Chapter Overview5.2. Methodology5.3. Assumptions and Key Parameters5.4. CRISPR-based Genome Editing Service Providers: Competitive Landscape5.4.1. Small-sized Companies5.4.2. Mid-sized Companies5.4.3. Large Companies

6. COMPANY PROFILES6.1. Chapter Overview6.2. Applied StemCell6.2.1. Company Overview6.2.2. Service Portfolio6.2.3. Recent Developments and Future Outlook6.3. BioCat6.4. Biotools6.5. Charles River Laboratories6.6. Cobo Scientific6.7. Creative Biogene6.8. Cyagen Biosciences6.9. GeneCopoeia6.10. Horizon Discovery6.11. NemaMetrix6.12. Synbio Technologies6.13. Thermo Fisher Scientific

7. PATENT ANALYSIS7.1. Chapter Overview7.2. Scope and Methodology7.3. CRISPR-based Genome Editing: Patent Analysis7.3.1. Analysis by Application Year and Publication Year7.3.2. Analysis by Geography7.3.3. Analysis by CPC Symbols7.3.4. Emerging Focus Areas7.3.5. Leading Players: Analysis by Number of Patents7.4. CRISPR-based Genome Editing: Patent Benchmarking Analysis7.4.1. Analysis by Patent Characteristics7.5. Patent Valuation Analysis

8. ACADEMIC GRANT ANALYSIS8.1. Chapter Overview8.2. Scope and Methodology8.3. Grants Awarded by the National Institutes of Health for CRISPR-based8.3.1. Year-wise Trend of Grant Award8.3.2. Analysis by Amount Awarded8.3.3. Analysis by Administering Institutes8.3.4. Analysis by Support Period8.3.5. Analysis by Funding Mechanism8.3.6. Analysis by Type of Grant Application8.3.7. Analysis by Grant Activity8.3.8. Analysis by Recipient Organization8.3.9. Regional Distribution of Grant Recipient Organization8.3.10. Prominent Project Leaders: Analysis by Number of Grants8.3.11. Emerging Focus Areas8.3.12. Grant Attractiveness Analysis

9. CASE STUDY: ADVANCED CRISPR-BASED TECHNOLOGIES/SYSTEMS AND TOOLS9.1. Chapter Overview9.2. CRISPR-based Technology Providers9.2.1. Analysis by Year of Establishment and Company Size9.2.2. Analysis by Geographical Location and Company Expertise9.2.3. Analysis by Focus Area9.2.4. Key Technology Providers: Company Snapshots9.2.4.1. APSIS Therapeutics9.2.4.2. Beam Therapeutics9.2.4.3. CRISPR Therapeutics9.2.4.4. Editas Medicine9.2.4.5. Intellia Therapeutics9.2.4.6. Jenthera Therapeutics9.2.4.7. KSQ Therapeutics9.2.4.8. Locus Biosciences9.2.4.9. Refuge Biotechnologies9.2.4.10. Repare Therapeutics9.2.4.11. SNIPR BIOME9.2.5. Key Technology Providers: Summary of Venture Capital Investments9.3. List of CRISPR Kit Providers9.4. List of CRISPR Design Tool Providers

10. POTENTIAL STRATEGIC PARTNERS10.1. Chapter Overview10.2. Scope and Methodology10.3. Potential Strategic Partners for Genome Editing Service Providers10.3.1. Key Industry Partners10.3.1.1. Most Likely Partners10.3.1.2. Likely Partners10.3.1.3. Less Likely Partners10.3.2. Key Non-Industry/Academic Partners10.3.2.1. Most Likely Partners10.3.2.2. Likely Partners10.3.2.3. Less Likely Partners

11. MARKET FORECAST11.1. Chapter Overview11.2. Forecast Methodology and Key Assumptions11.3. Overall CRISPR-based Genome Editing Services Market, 2019-203011.4. CRISPR-based Genome Editing Services Market: Distribution by Regions, 2019-203011.4.1. CRISPR-based Genome Editing Services Market in North America, 2019-203011.4.2. CRISPR-based Genome Editing Services Market in Europe, 2019-203011.4.3. CRISPR-based Genome Editing Services Market in Asia Pacific, 2019-203011.4.4. CRISPR-based Genome Editing Services Market in Rest of the World, 2019-203011.5. CRISPR-based Genome Editing Services Market: Distribution by Type of Services, 2019-203011.5.1. CRISPR-based Genome Editing Services Market for gRNA Construction, 2019-203011.5.2. CRISPR-based Genome Editing Services Market for Cell Line Engineering, 2019-203011.5.3. CRISPR-based Genome Editing Services Market for Animal Model Generation, 2019-203011.6. CRISPR-based Genome Editing Services Market: Distribution by Type of Cell Line, 2019-203011.6.1. CRISPR-based Genome Editing Services Market for Mammalian Cell Lines, 2019-203011.6.2. CRISPR-based Genome Editing Services Market for Microbial Cell Lines, 2019-203011.6.3. CRISPR-based Genome Editing Services Market for Other Cell Lines, 2019-2030

12. SWOT ANALYSIS12.1. Chapter Overview12.2. SWOT Analysis12.2.1. Strengths12.2.2. Weaknesses12.2.3. Opportunities12.2.4. Threats12.2.5. Concluding Remarks

13. EXECUTIVE INSIGHTS

14. APPENDIX 1: TABULATED DATA

15. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

Companies Mentioned

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It may finally be a happy Christmas for a brave toddler recovering from a rare combination of cancers after pioneering stem cell treatment.

Amelia Topa, who celebrated her second birthday yesterday, is looking forward to spending the festive period with her family at home in Turriff.

Her parents Kerri Paton, 23, and Igor Topa, 24, were told that raised purple spots across Amelias body could be a sign of something seriously wrong when she was born at Dr Grays Hospital in Elgin.

Amelia was soon after diagnosed with a mix of two types of leukemia acute lymphoblastic leukaemia and acute myeloid leukaemia and spent Christmas in hospital.

Miss Paton said: Its rare enough to be born with leukaemia but to be born with a mix of two kinds is almost unheard of.

Doctors gave Amelia a bone marrow transplant using stem cells donated by a managed between 16 and 30.

The treatment worked and, by the following autumn, she was home and awaiting the arrival of her baby brother.

But tragedy struck when Amelias grandmother, Angela McNabb, died suddenly from heart failure aged 48 the day before Amelias birthday.

Miss Paton said: My mum was my best friend, she was everything to me.

Mum absolutely loved Amelia and was so close to her.

My major source of support was gone and I hadnt even had the chance to say goodbye. I couldnt believe it. It was so unfair. Last Christmas was heartbreaking.

Things went from bad to worse for the family in February, when tests showed that Amelias cancer had returned.

After intense chemotherapy she was given a second transplant using stem cells from umbilical cord blood flown specially from America at the end of June.

That procedure was a success and the toddler has entered remission.

Having spent Christmas in 2017 in hospital, and in mourning last year, Amelias parents are now looking forward to a happy festive season.

Miss Paton said: Amelia soared through the transplant and shes doing really well now,I couldnt be prouder.

I hope Amelias story will help other families going through cancer there is a light at the end of the tunnel.

Amelia has now been selected to receive the first Cancer Research UK children and young people star award in recognition of the courage she showed since being diagnosed.

The award, supported by TK Maxx, is open to all people under 18s who currently have cancer or who have been treated in the last five years with every child being awarded a trophy, TK Maxx gift card, t-shirt and certificate signed by the likes of Nanny McPhee star Dame Emma Thompson.

Spokeswoman for the charity, Lisa Adams, said: We know that a cancer diagnosis is devastating at any age, but that it can be particularly difficult for a child or young person and their families.

Thats why were calling on families across Scotland to nominate inspirational youngsters for an award so that we can recognise their incredible courage.

Nominations can be made online at cruk.org/childrenandyoungpeople

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North-east toddler overcomes cancer after pioneering stem cell treatment - Press and Journal

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Angie Grice is a such huge Clemson football fan that in 20 years shes rarely missed a home game or any of the Tigers-Gamecocks match-ups.

At her tailgate parties, the plates, the tablecloth and even a rug are orange.

Angie Grice gets a visit from the Clemson Tigers mascot during her three months in the hospital.(Photo: Bon Secours St. Francis Health System)

But for this years annual Thanksgiving weekend face-off between Clemson and USC, shell be watching from home.

Diagnosed with aplastic anemia in May, the Simpsonville woman spent three months in the hospital and is still too sick to cheer her beloved Tigersfromthe stadium. Instead, she hopes to have a few friends over to catch the gameon TV.

Ive liked Clemson forever," she told The Greenville News.

"Im missing the game this yearand Im sad about that, she said.But its OK. At least Im able to watch it.

Grice, 52, first realized something was wrong in August 2018 when she suddenly had trouble crossing the parking lot from her car to her job as a physical therapy assistant.

I was very short of breath, she recalls. It would take me a long time to do anything. I just couldnt breathe.

She saw her family doctor, who sent her to Bon Secours St. Francis Health System when her blood work wassuspicious.

Angie Grice at Clemson University(Photo: Angie Grice)

An initial bone marrow biopsy was negative.But a second revealedaplastic anemia,which prevents the bone marrow frommakingenough new blood cells for the body to function normally, according to the National Institutes of Health.

The condition is so rare it strikes only 600 to 900 Americansa year, according to the The Aplastic Anemia and MDS International Foundation.

Symptoms include fatigue, weakness, dizziness, shortness of breath, infections, and easy bruising or bleeding,the NIHreports.The cause can bethe bodys own immune system attackingthe bone marrow, heredity, some drugs, and certain toxins likepesticides and benzene.

When St. Francis hematologist Dr. Fahd Quddus first saw her, Grices platelet level was 8,000 compared to a normal of 150,000.

Whenever you drop below 20,000, youre at risk of significant, life-threatening bleeding, he said. She also had significant anemia. And her white cells were also very low.

She was started on immunosuppressive medication and other drugs in combination with blood transfusions. But sadly, he said, she suffered multiple infections, fevers and a mild stroke, requiring her to stay in the hospital.

Dr. Fahd Quddus(Photo: Bon Secours St. Francis Health System)

For a few weeks, it was touch and go, Quddus said. She was very sick.

Grice'sblood counts eventually rebounded and though shes now out of the hospital, shestill needsregulartransfusions.

She's wellenough to begin a new treatment, he said, butnot yet strong enoughfor a stem celltransplant.

Theresstill a long road to recovery, Quddussaid. But she always looks at it half full. And thats a good thing because people who stay positive can do better.

No longer able to work because of the weakness and danger of infection, Gricesays shes doing OK thanks tofamily and friends.

Angie Grice at a Clemson game(Photo: Angie Grice)

My mom and dad and sister help, she says. And I am truly blessed with a lot of friends who help.

In years past, Grice and her friends arrived at the stadiumseveral hours before kick-off, spending 10 to 12 hours thereon game days.

Inside their orange tent, they set up a coupleTVs to watch other games before and after the Clemson game. There was always plenty ofgood food,smack talk and Tigersmerchandise.

Were a little over the top, she says. But its fun.

During her grueling three-monthhospital stay, it was a visit from the Clemson Tigers mascot that lifted her spirits.

One of Angie Grice's many Clemson decorations(Photo: Angie Grice)

While watching from home wont be as exciting, Grice says shes going to make the best of it. And when asked whos going to win this years game, sheexclaims, Clemson, of course!

If you ask Carolina, they will say they are, she adds with a chuckle. But theyre delusional. Were going to win this year.

Read or Share this story: https://www.greenvilleonline.com/story/news/local/2019/11/28/clemson-fan-sidelined-illness-watch-carolina-game-home/4155439002/

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Despite illness, this Clemson fan will be tuned in for the Tigers-Gamecocks game - Greenville News

Bone Marrow Stem Cells Comments Off on Despite illness, this Clemson fan will be tuned in for the Tigers-Gamecocks game – Greenville News | November 28th, 2019

(MENAFN - The Conversation) The global AIDS response has made significant progress in reducing HIV infections and AIDS-related deaths. New HIV infections dropped by16%from 1.9 million 2010 to 1.6 million in 2017. And the number of AIDS-related deaths decreased from 1.4 million to 940 000 in the same period.

But HIV/AIDS has not been brought under control and new infections continue to drive the epidemic. AIDS remains a leadingcause of deathin Africa.

Even if new infections are prevented,36.9 millionpeople with HIV around the world must take antiretroviral treatment to live a healthy life. While treatment is now as simple as taking a single pill a day, there are still many challenges to daily adherence, including ongoing stigma.

An ultimate solution would be a workable cure. At the recent Conference on Retroviruses and Opportunistic Infections researchersconfirmedthe second ever case of HIV remission or 'cure'. Known as the 'London patient', the person went into remission after a stem cell transplant as part of his treatment for cancer. He emerged from the procedure free of both his life-threatening Lymphoma and need for anti-HIV therapy.

The'Berlin patient' , Timothy Brown, made global headlines in 2008 when scientists announced that he had been cured of HIV. It's been 12 years since Brown was cured, after undergoing chemotherapy, total body irradiation and two stem cell transplants. Brown has been off treatment since the transplant and, after multiple tissue sampling procedures, has no remaining evidence of HIV reservoirs. The London patient is now the longest adult HIV remission after stem cell transplantation since the 'Berlin patient'.

This development is a triumph for medical science as well as for the London patient. But, as exciting as it is, stem cell transplant is a gruelling and dangerous procedure and isn't the magic bullet that will end HIV/AIDS. This is because it's unfortunately not a scalable, feasible cure for the 39 million people currently living with HIV.

The 'London patient' was HIV positive, but it was his Hodgkin's lymphoma that led to the need for a stem cell transplant.

The HI virus must link to a human host T cell in the blood or lymph nodes to replicate and infect the body. The virus attaches itself to a set of special links on the human T cell. If one of those links isn't available due to genetic mutations, the virus may find it harder to get an infection foothold.

One such genetic mutation occurs in a link called the 'CCR5 receptor'. Some people have this mutation naturally. The 'London patient', while on antiretroviral therapy and virally suppressed, had a bone marrow transplant as part of his lymphoma treatment. The bone marrow donor had the genetic mutation and passed it on to the 'London patient' through the procedure, making it more difficult for HIV to replicate.

The 'London patient' stopped taking antiretroviral therapy 16 months after the transplant. And 18 months later the virus remains undetectable. Usually, when a person with HIV stops treatment, the virus rebounds within the first month.

The achievement of remission in a second patient has provided further critical information to inform our understanding of how HIV infection occurs and the interaction between human cells and the virus.

As important as this work is, there's no scalable cure yet and it's also vital that researchers and countries keep putting effort into prevention. Important work continues to be done in this area.

As HIV cure research goes on, so does research into HIV prevention tools, such asPre-exposure prophylaxis(a daily pill that protects you from HIV infection) and the development of apreventative vaccine .

Two late stage vaccinetrialsare underway in sub-Saharan Africa. Results will be available in 2022. A preventative vaccine would also greatly enhance efforts to being the HIV epidemic under control.

A working cure, together with a preventative vaccine would be the ingredients for HIV eradication. Until then we need to get effective, accessible treatment for all who need it, while deploying the many prevention tools at our disposal.

MENAFN2811201901990000ID1099339725

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Why ending HIV still rests on a working cure -- as well as prevention - MENAFN.COM

Bone Marrow Stem Cells Comments Off on Why ending HIV still rests on a working cure — as well as prevention – MENAFN.COM | November 28th, 2019

Eileen Booker sat in her Southern California home last Sunday night, watching the Packers game like she does every week.

She grew up in Green Bay, and her sister still lives there. Her parents bought season tickets in 1957 and her father never missed a home game. She remembers sticking to frigid metal bleachers as a kid until the clock showed zeroes in the fourth quarter, win or lose, and always burning her lips with hot chocolate.

Still a die-hard fan today, Eileen was glued to her television for a prime-time game between the Packers and 49ers, even as her favorite team trailed, 10-0, early in the second quarter.

She had no idea her name was about to be plastered on TV screens across America.

After Packers outside linebacker ZaDarius Smith sacked 49ers quarterback Jimmy Garoppolo deep in 49ers territory, he immediately found the nearest camera and lifted his jersey, revealing a white undershirt that read, WE ...

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How the Packers' Za'Darius Smith brought joy and awareness to one woman's cancer fight - The Athletic

Bone Marrow Stem Cells Comments Off on How the Packers’ Za’Darius Smith brought joy and awareness to one woman’s cancer fight – The Athletic | November 28th, 2019

A handful of biotech companies have been laboring for years to turn stem cells into treatments that can repair damaged tissue after a heart attack, but with limited success. A team from the Cincinnati Children's Hospital Medical Center tracked stem cells injected into the hearts of mice, and what they found could explain why this particular attempt at regenerative medicine has not proven effectiveand inspire new ideas for repairing damaged heart tissue.

The researchers injected both live and dead heart stem cells into mice with damaged hearts and discovered that the procedure touched off extreme inflammation. That inflammatory response generated a healing process, which in turn improved the mechanical properties of the injured area, they reported in the journal Nature.

During the study, the scientists used two types of stem cells that have been tested for the treatment of damaged heart tissue in clinical trials: cardiac progenitor cells and bone marrow mononuclear cells. They also tried injecting the chemical zymosan, which has been shown to prompt an immune response that can promote healing.

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All three treatments activated macrophage cells from the immune system, which helped the animals hearts heal with a more optimized scar and improved contractile properties, said lead investigator Jeffery Molkentin, Ph.D., director of Molecular Cardiovascular Microbiology at Cincinnati Children's Hospital Medical Center and professor at the Howard Hughes Medical Institute, in a statement.

Problem is, the initial goal of injecting stem cells into patients with damaged hearts was to regenerate cardiomyocytes. The Cincinnati team had previously reported that c-kit+ cardiac progenitor cells only produce tiny amounts of new cardiomyocytesnot nearly enough to provide any therapeutic value.

This new study validated that finding, leading Molkentin and colleagues to propose that cardiac researchers re-evaluate the current planned cell therapy based clinical trials to ask how this therapy might really work.

Whats more, the researchers found that stem cells and zymosan were only effective if they were injected directly into mouse hearts in the areas where the damage had occurred. This approach is at odds with most stem cell clinical trials, which involve infusing cells into the circulatory system.

Our results show that the injected material has to go directly into the heart tissue flanking the infarct region. This is where the healing is occurring and where the macrophages can work their magic, Molkentin said.

RELATED: Stem cell combo repairs damaged hearts in rats

The findings from the Cincinnati team could prove valuable in a field that has seen its share of disappointments. Australia-based Mesoblast, for one, released results from a phase 2 trial last year that showed patients who received injections of mesenchymal precursor cells did not improve to the point where they could stop using their left ventricular assist devices.

And the National Heart, Lung, and Blood Institute of the National Institutes of Health had to halt a trial testing c-kit+ cells and mesenchymal stem cells in patients with heart failure because of safety concerns.

The Cincinnati Children's Hospital researchers believe their findings could inspire new regenerative approaches to treating heart disease. They are now planning further studies focused on harnessing the healing power of macrophages.

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Stem cells don't repair injured hearts, but inflammation might, study finds - FierceBiotech

Cardiac Stem Cells Comments Off on Stem cells don’t repair injured hearts, but inflammation might, study finds – FierceBiotech | November 28th, 2019

Recently, a new study that appears in the journal Nature, focuses on stem cell therapy and shows unexpected ways in which it may be helpful in recovering the health of the heart. Stem cell therapy has become popular in the past few years due to its benefits for a big number of health conditions.

Currently, there is major ongoing research on stem cells since they are responsible for the regeneration of new cells and may play a fundamental role in understanding the development of a variety of different diseases as well as their potential treatments.

Some of the recent discoveries of medical science include using stem cells as regenerative medicine as they can be turned into particular types of cells that may be able to replace tissues damaged as a result of health issues and thereby control the disease.

Read also:New Study Reveals Hydromethylthionine Slows Cognitive Decline and Brain Atrophy

The therapy can be specifically useful for people with conditions such as type 1 diabetes, spinal cord injuries, Alzheimers disease, Parkinsons disease, stroke, cancer, burns, amyotrophic lateral sclerosis, heart disease, and osteoarthritis.

At the moment, the most successful procedure that involves stem cell therapy is performing a bone marrow transplant. This surgical operation replaces the cells which have been damaged during chemotherapy by programmed stem cells. People are usually able to maintain and live a normal life after recovery from the surgery.

Furthermore, stem cell usage in clinical trials designed for testing the effectiveness, safety, and potential negative impact of new drugs. To do so, the stem cells can be programmed into becoming the type of cells that the drug aims to target.

The new study, which was led by Jeffery Molkentin who is a professor of the Howard Hughes Medical Institute (HHMI) and the director of Molecular Cardiovascular Microbiology a Cincinnati Childrens Hospital Medical Center, takes data from a study from the same journal, Nature, from the years 2014 which was conducted by the same medical team.

In the new paper, the team with Molkentin as the principal investigator found some unexpected results. There were two types of stem cells in the clinical trial cardiac progenitor cells and bone marrow mononuclear cells.

The main objective of the new trial was to re-evaluate the results of the 2014 study, which showed that injecting c-kit positive heart stem in the heart does not help in the regeneration of cardiomyocytes, to see how the cell therapy can be made to be effective.

It was instead discovered that injecting an inert chemical called zymosan, which is designed particularly for inducing an innate immune response, or dead stem cells can also be beneficial for the recovery of heart as they may speed up the healing procedure.

Injecting either dead stem cells or zymosan led to a reduction in the development of cellular matrix connective tissue in the areas which had been damaged in the heart. In addition, the mechanical properties of the targeted scar also improved.

Another important finding was that chemical substances such as zymosan are required to be injected directly into the heart for optimum results. In previous clinical trials, direct injections were avoided for safety reasons.

Molkentin and the team state that follow-up studies and trials on this new discovery are imminent as they may be important for developing therapies in the future.

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Stem Cell Therapy May Improve Heart Health In New Ways - TheHealthMania

Cardiac Stem Cells Comments Off on Stem Cell Therapy May Improve Heart Health In New Ways – TheHealthMania | November 28th, 2019

Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

GREENVILLE, S.C.Hailie and Treylin Hyman saw the bruising on their baby girls leg as a sign that the active 1-year-old was learning to walk.

But as a blood test would later reveal, little Maci was actually suffering from an extremely rare blood cancer that threatened her life without a risky treatment - atreatmentalmost as dangerous as the disease.

In the beginning, it was very scary, Hailie Hyman told The Greenville News.

I couldnt think of anything but the bad things, she confessed. It was all about the statistics. And the statistics arent good.

Terrifying months followed the diagnosis, punctuated by one critical complication after another, leaving the Boiling Springs couple to wonder if Maci would survive.

Somehow, though, the blue-eyed toddler pulled through.And now her family is looking forward to a special Thanksgiving with much to be grateful for.

Alyssa Carson is 18 and has a pilot's license: She wants to be in the crew that colonizes Mars

The Hymans journey began last February atMacis 1-year-old well-child checkup.

We had no idea anything was wrong, her mom said.But they did a routine (blood test) and a couple of hours later, we got a call saying her platelets were very low.

The Hymans were referred to a hematologist who found other abnormalities in Macis blood and scheduled a bone marrow biopsy to investigate further.

Hailie Hyman holds her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

During the procedure, the child suffered an aneurysm in an artery and went into cardiac arrest. The team performed CPR on her for 20 minutes before she was stabilized, her mom said.

Later, in the pediatric intensive care unit, she suffered internal bleeding, too.

It was really hard, she said. There were many nights that I would just pray and pray and pray.

Initially believing Maci had leukemia, doctors subsequently determined she had myelodysplastic syndrome, or MDS.

The condition occurs when abnormal cells in the bone marrow leave the patient unable to make enough blood, according to the American Cancer Society.

Its rare, afflicting as few 10,000 Americans a year, though the actual number is unknown.

Maci Hyman, 1, interacts with hospital staff before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

In children, its rarer still. Most people arediagnosed in their 70s.

We were told that just four out of 1 million children get it every year, Hailie Hyman said.

That made the diagnosis elusive at first, said Dr. Nichole Bryant, a pediatric hematologist-oncologist with Prisma Health-Upstate, formerly Greenville Health System.

Shes the only one Ive seen in my career, she said.

Maci had to have regular blood transfusions, antibiotics and other medications to fight the MDS, Bryant said. But the only hope for a cure was a stem cell transplant at the Medical University of South Carolina in Charleston.

When they said that was the only treatment plan for MDS, I of course went to Google, Hailie Hyman said. I read about transplant patients and ...all the complications. It was terrifying. But no matter how many bad things I saw, we had to do it. There is no other option.

The transplantis extremely risky.

Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

First, high doses of chemotherapy are given to destroy the diseased bone marrow, leaving the patient without an immune system, so fighting infections becomes a challenge. Then healthy donor marrow is infused.

Its also fraught with potentially life-threatening complications, including graft vs. host disease, which occurs when immune cells from the donor attack the patients body, Bryant said. Other complications include permanent kidney damage and gastrointestinal problems.

They have to go to hell and back, she said. But its the only option for long-term survival.

Maci had a really rough start, suffering lots and lots and lots of complications, Bryant said.

Her kidneys failed, so she wound up on dialysis. When she couldnt breathe on her own, she was put on a ventilator. And because she couldnt eat, she had to be tube fed.

Hailie Hyman looks at a fish tank with her daughter Maci, 1, before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

She had blistering sores in her mouth and throughout her GI tract, her mom said. Because her liver wasnt functioning properly, her abdomen filled up with fluid that had to be drained. She was bleeding so profusely in her lungs that one of them collapsed.

Maci, who was sedated through much of it, was put on full life support, she said.

That night we almost lost her, her mom said. We were in the hallway crying our eyes out. We didnt know what do to or think. It was pretty scary for a while.

Somehow, Maci made it.

There were so many times during her first months that it seemed like she would not survive, Bryant said. So the fact that she is here ... is really a miracle.

Macis family found an unrelated donor through the National Marrow Donor Program, enlisting hundreds of other people to join the registry in the process, Bryant said.

Nichole Bryant, M.D.(Photo: Provided)

It was an important part of their journey that maybe didnt directly benefit Maci, she said. But if everybody did that, we wouldnt have difficulty finding a donor for anybody.

Doctors have no explanation for why Maci got MDS. She didnt carry the genetic mutation for it and there is no family history.

She is a rare child - and not in a good way, her mom said, adding,Youve got to laugh sometimes or youre going to cry.

A dying man wanted one last beer with his sons: The moment resonated with thousands

Maci was admitted to MUSC on June 2 and released on Oct. 14.

The Hymans, both 22, spent the entire time in Charlestonwhile Hailies mom cared for their older daughter, Athena, now 2.

Treylins employer held his welding job open for him. And other friends and family members did what they could to help.

We had many, many people very generously donate to us to cover expenses at home and living expenses where we were, Hailie Hyman said.

We are thankful for everyone who helped us through it the cards, the gifts, the donations. Every single cent is greatly appreciated.

Maci's doing well, but recovery from a transplant can take months to years, Bryant said.

Her kidneys are functioning again so she was able to come off dialysis. But she still must take many medications, including anti-rejection drugs that suppress her immune system and leaveher at risk for infection. And she still must be tube fed.

She is miles ahead of where she was two months ago, Bryant said. But she still has a long way to go. Its a long, long road.

Macis mom says she can be up and playing one day and flopped over on the couch another. She still experiences a lot of nausea and vomiting, but is doing well compared to where she was.

Hailie Hyman pulls her daughter Maci, 1, in a wagon in the hallway before an appointment at the Prisma Health Pediatric Hematology Oncology Center Monday, Nov. 4, 2019.(Photo: JOSH MORGAN/Staff)

So as the nation pauses to give thanks this Thanksgiving, she says the family will be countingtheir many blessings family andfriends, Gods mercy, andthe doctors and nurses who saved Macis life.

She has battled a lot and overcome a lot, she said. I have no doubt she will be able to get through.

Want to know more about becoming a marrow donor? Go to bethematch.org.

Follow Liv Osby on Twitter:@livgnews

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Read or Share this story: https://www.usatoday.com/story/news/health/2019/11/28/south-carolina-toddler-survives-rare-blood-cancer-risky-procedure/4321002002/

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South Carolina toddler survives rare cancer and the risky procedure used to treat it - USA TODAY

Cardiac Stem Cells Comments Off on South Carolina toddler survives rare cancer and the risky procedure used to treat it – USA TODAY | November 28th, 2019

HONG KONG, Nov. 26, 2019 /PRNewswire/ --Novoheart("Novoheart" or the "Company") (TSXV: NVH; FWB: 3NH), a global stem cell biotechnology company, is pleased to announce a collaboration with global biopharmaceutical company AstraZeneca, in an effort to develop the world's first human-specific in vitro, functional model of heart failure with preserved ejection fraction (HFpEF), a common condition especially among the elderly and in women, with the reported prevalence approaching 10% in women over the age of 80 years.[1]

Heart failure (HF) is a global pandemic with an estimated 64.3 million cases worldwide in 2017, with an increasing trend in prevalence[2]. The annual global economic burden of HF is estimated at over US$100 billion[3]. Accounting for approximately 50% of HF cases, HFpEF in particular is a major and growing public health problem worldwide, with its pathological mechanisms and diverse etiology poorly understood. Due to these complexities, models of the disease available to date, including various animal models, have limited ability to mimic the clinical presentation of HFpEF[4]. Therefore, drug developers lack an effective tool for preclinical testing of drug candidates for efficacy, and as a result, clinical outcomes for HFpEF have not improved over the last decades, with no effective therapies available.

In collaboration with the Cardiovascular, Renal and Metabolism therapy area of AstraZeneca, the initial phase of the project aims to establish a new in vitro model, leveraging Novoheart's proprietary 3-D human ventricular cardiac organoid chamber (hvCOC) technology, that reproduces key phenotypic characteristics of HFpEF. Also known as "human heart-in-a-jar", the hvCOC is the only human engineered heart tissue available on the market to date that enables clinically informative assessment of human cardiac pump performance including ejection fraction and developed pressure. Unlike animal models, engineered hvCOCs can be fabricated with specific cellular and matrix compositions, and patient-specific human induced pluripotent stem cells (iPSCs), that allow control over their physical and mechanical properties to mimic those observed in HFpEF patient hearts. Together with Novoheart's proprietary hardware and software, this aims to provide a unique assay for understanding the mechanisms of HFpEF, identification of new therapeutic targets, and assessment of novel therapeutics for treating HFpEF patients. Novoheart will exclusively own the intellectual property rights to the newly developed HFpEF hvCOC model.

"We are delighted to partner with AstraZeneca, an organization which has long invested in cardiovascular research and is committed to bringing new therapeutic solutions to patients with heart failure," said Novoheart CSO, Dr. Kevin Costa. "We look forward to co-developing this new HFpEF hvCOC model into a powerful new tool in the worldwide battle against heart failure."

Regina Fritsche Danielson, Senior Vice President, Head of Research and Early Development, Cardiovascular, Renal and Metabolism, BioPharmaceuticals R&D, AstraZeneca, said, "There are significant unmet treatment needs in patients with heart failure with preserved ejection fraction. By combining Novoheart's proprietary hvCOC model with our expertise in heart failure, we aim to create the first in vitro model reproducing phenotypic characteristics of heart failure with preserved ejection fraction. This could bridge the gap between in vivo animal models and clinical trials to help accelerate the drug discovery process by providing human-specific preclinical data."

[1] Heart Fail Clin. 2014; 10(3):377-388.

[2] Lancet. 2018; 392:1789-1858.

[3] Int J Cardiol. 2014; 171(3):368-76.

[4]JACC Basic Transl Sci. 2017; 2(6):770-789.

About Novoheart:

Novoheartis a global stem cell biotechnology company pioneering an array of next-generation human heart tissue prototypes. It is the first company in the world to have engineered miniature living human heart pumps that can revolutionize drug discovery, helping to save time and money for developing new therapeutics. Also known as 'human heart-in-a-jar', Novoheart's bio-artificial human heart constructs are created using state-of-the-art and proprietary stem cell and bioengineering approaches and are utilized by drug developers for accurate preclinical testing of the effectiveness and safety of new drugs, maximizing the successes in drug discovery whilst minimizing costs and harm caused to patients. With the recent acquisition of Xellera Therapeutics Limited for manufacturing Good Manufacturing Product (GMP)-grade clinical materials, Novoheart is now developing gene- and cell-based therapies as well as next-generation therapeutics for cardiac repair or regeneration.

Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

Cautionary Note Regarding Forward-Looking Statements

Information set forth in this news release may involve forward-looking statements under applicable securities laws. Forward-looking statements are statements that relate to future, not past, events. In this context, forward-looking statements often address expected future business and financial performance, and often contain words such as "anticipate", "believe", "plan", "estimate", "expect", and "intend", statements that an action or event "may", "might", "could", "should", or "will" be taken or occur, or other similar expressions. By their nature, forward-looking statements involve known and unknown risks, uncertainties and other factors which may cause the actual results, performance or achievements, or other future events, to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Such factors include, among others, the risks identified in under the heading "Risk Factors" in Novoheart's annual information form for the year ended June 30, 2018 or other reports and filings with the TSX Venture Exchange and applicable Canadian securities regulators. Forward-looking statements are made based on management's beliefs, estimates and opinions on the date that statements are made and the respective companies undertakes no obligation to update forward-looking statements if these beliefs, estimates and opinions or other circumstances should change, except as required by applicable securities laws. Investors are cautioned against attributing undue certainty to forward-looking statements.

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Novoheart to Co-develop First of its Kind Human Heart-in-a-Jar Model of Heart Failure with AstraZene - PharmiWeb.com

Cardiac Stem Cells Comments Off on Novoheart to Co-develop First of its Kind Human Heart-in-a-Jar Model of Heart Failure with AstraZene – PharmiWeb.com | November 28th, 2019