A New CRISPR Tool Flips Genes On and Off Like a Light Switch – Singularity Hub
By daniellenierenberg
CRISPR is revolutionary. Its also a total brute.
The classic version of the gene editing wunderkind literally slices a gene to bits just to turn it off. Its effective, yes. But its like putting an electrical wire through a paper shredder to turn off a misbehaving light bulb. Once the wires are cut, theres no going back.
Why not add a light switch instead?
This month, a team from the University of California, San Francisco (UCSF) reimagined CRISPR to do just that. Rather than directly acting on genesirrevocably dicing away or swapping genetic lettersthe new CRISPR variant targets the biological machinery that naturally turns genes on or off.
Translation? CRISPR can now flip a light switch to control geneswithout ever touching them directly. It gets better. The new tool, CRISPRoff, can cause a gene to stay silent for hundreds of generations, even when its host cells morph from stem cells into more mature cells, such as neurons. Once the sleeping beauty genes are ready to wake up, a complementary tool, CRISPRon, flips the light switch back on.
This new technology changes the game so now youre basically writing a change [into genes] that is passed down, said author Dr. Luke Gilbert. In some ways we can learn to create a version 2.0 of CRISPR-Cas9 that is safer and just as effective.
The crux is something called epigenetics. Its a whole system of chemicals and proteins that controls whether a gene is turned on or off.
If that sounds confusing, lets start with what genes actually look like inside a cell and how they turn on. By turning on, I mean that genes are made into proteinsthe stuff that builds our physical form, controls our metabolism, and makes us tick along as living, breathing humans.
Genes are embedded inside DNA chains that wrap very tightly around a core proteinkind of like bacon-wrapped asparagus. For genes to turn on, the first step is that they need a bunch of proteins to gently yank the DNA chain off the asparagus, so that the genes are now free-floating inside their cellular space capsule, called the nucleus.
Once that chunk of bacon-y DNA is free, more proteins rush over to grab onto the gene. Theyll then roll down the genes nucleotides (A, T, C, and G) like a lawn mower. Instead of mulch, however, this biological machine spews out a messenger that tells the cell to start making proteinsmRNAs. (Yup, the same stuff that makes some of our Covid-19 vaccines.) mRNA directs our cells protein factory to start production, and voil, that gene is now turned on!
Anything that disrupts this process nukes the genes ability to turn into proteins, essentially shutting it off. Its enormously powerfulbecause one single epigenetic machine can control hundreds or thousands of genes. Its a master light switch for the genome.
The team started with a CRISPR system that has a neutered Cas9. This means that the protein normally involved in cutting a gene, Cas9, can no longer snip DNA, even when tethered to the correct spot by the other component, the guide RNA bloodhound. They then tacked on a protein thats involved in switching off genes to this version of CRISPR.
Heres the clever part: the protein is designed to hijack a natural epigenetic process for switching genes off. Genes are often shut down through a natural process called methylation. Normally, the process is transient and reversible on a gene. CRISPRoff commandeers this process, in turn shutting down any targeted gene but for a far longer period of timewithout physically ripping the gene apart.
Thanks to epigenetics enhancing power, CRISPRoff lets researchers go big. In one experiment targeting over 20,000 genes inside immortalized human kidney cells with CRISPRoff, the team was able to reliably shut those genes off.
Not satisfied with a one-way street, the team next engineered a similar CRISPR variant, with a different epigenetics-related protein, dubbed CRISPRon. In cells inside petri dishes, CRISPRon was able to override CRISPRoff, and in turn, flip the genes back on.
We now have a simple tool that can silence the vast majority of genes, said study author Dr. Jonathan Weissman. We can do this for multiple genes at the same time without any DNA damage and in a way that can be reversed.
Even crazier, the off switch lasted through generations. When the team turned off a gene related to the immune system, it persisted for 15 monthsafter about 450 cellular generations.
The edits also lasted through a fundamental transformation, that is, a cells journey from an induced pluripotent stem cell (iPSC) to a neuron. iPSCs often start as skin cells, and are rejuvenated into stem cells through a chemical bath, when they then take a second voyage to become neurons. This process often wipes away epigenetic changes. But to the authors surprise, CRISPRoffs influence remained through the transformations. In one experiment, the team found that shutting off a gene related to Alzheimers in iPSCs also reduced the amount of subsequently encoded toxic proteins in the resulting neurons.
What we showed is that this is a viable strategy for silencing Tau and preventing that protein from being expressed, said Weissman, highlighting just one way CRISPRoffand controlling the epigenome in generalcan alter medicine.
This isnt the first time someones tried to target the epigenome with CRISPR. The same team previously experimented with another set of CRISPR variants that tried the same thing. The difference between the two is time and stability. With the previous setup, scientists struggled to keep the light switch off for a single generation. The new one has no trouble maintaining any changes through multiple divisionsand transformationsin the genome.
A reliable CRISPR tool for epigenetics is insanely powerful. Although we have drugs that work in similar ways, theyre far less accurate and come with a dose of side effects. For now, however, CRISPRoff and CRISPRon only work in cells in petri dishes, and the next step towards genomic supremacy would be to ensure they work in living beings.
If thats the case, it could change genetic editing forever. From reprogramming biological circuits in synthetic biology to hijacking or reversing ones to prevent disease, epigenetic reprogramming offers a way to do it all without ever touching a gene, nixing the threat of mutationswhile leading to lasting effects through generations.
I think our tool really allows us to begin to study the mechanism of heritability, especially epigenetic heritability, which is a huge question in the biomedical sciences, said study author Dr. James Nuez.
Image Credit: nobeastsofierce/Shutterstock.com
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A New CRISPR Tool Flips Genes On and Off Like a Light Switch - Singularity Hub
Key Trends of Autologous Stem Cell Based Therapies Market 2021 Business Opportunities, Market Dynamics, Growth Size and Forecasts to 2026 – Clark…
By daniellenierenberg
New research report on Autologous Stem Cell Based Therapies market size by In4Research provides the latest developments in the market has attained and the latest news regarding the market and its building blocks. Some of the most accurate topics mentioned in the report include market drivers and restraints, challenges, opportunities, COVID-19 information related to the market, key players of the market, and the entire market segmentation.
This report provides data for the base year 2020 as well as the forecast period (2021-2026) along with the CAGR. This report contains detailed research of the market from the industry level to the market players and how the market is functioning at present along with all the relevant data and information. Thereby, the Autologous Stem Cell Based Therapies research report has been formulated encompassing all the key points that are present in the form of tables and graphs to make it more specific for decision-makers.
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The segmental analysis offered in the report pinpoints key opportunities available in the Autologous Stem Cell Based Therapies market through leading segments. The regional study of the Autologous Stem Cell Based Therapies market included in the report helps decision-makers to gain a sound understanding of the development of different geographical markets in recent years and going forth.
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To comprehend Global Autologous Stem Cell Based Therapies market dynamics in the world mainly, the worldwide Autologous Stem Cell Based Therapies market is analyzed across major regions. A customized study by region and country can be provided considering the below splits.
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The report provides in-depth information about profitable showing markets and examines the markets for the global Autologous Stem Cell Based Therapies market with business strategies of the key players and the new entering market industries are considered in detail and it presents the deep-dive eyesight of this Autologous Stem Cell Based Therapies market from 2021 to 2026 and prospective prediction market trends.
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Key Trends of Autologous Stem Cell Based Therapies Market 2021 Business Opportunities, Market Dynamics, Growth Size and Forecasts to 2026 - Clark...
Stem Cell Therapy Market worth $401 million by 2026 – Exclusive Report by MarketsandMarkets – PRNewswire
By daniellenierenberg
CHICAGO, April 28, 2021 /PRNewswire/ -- According to the new market research report "Stem Cell Therapy Marketby Type (Allogeneic, Autologous), Therapeutic Application (Musculoskeletal, Wound & Injury, CVD, Autoimmune & Inflammatory), Cell Source (Adipose tissue, Bone Marrow, Placenta/Umbilical Cord) - Global Forecasts to 2026", published by MarketsandMarkets, the global market is projected to reach USD 401 million by 2026 from USD 187 million in 2021, at a CAGR of 16.5% during the forecast period.
Browse and in-depth TOC on"Stem Cell Therapy Market"142 - Tables45 - Figures160 - Pages
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The Market growth is driven mainly by factors such as increasing investment in stem cell research and the rising number of GMP-certified stem cell manufacturing plants. However, factors such as ethical concerns and the high cost of stem cell research and manufacturing process likely to hinder the growth of this market.
The adipose tissue-derived MSCs segment accounted for the largest share of the cell source segment in the Stem Cell Therapy Market in 2020.
Based on the cell source from which stem cells are obtained, the global market is segmented into four sources. These include adipose tissue-derived MSCs (mesenchymal stem cells), bone marrow-derived MSCs, placenta/umbilical cord-derived MSCs, and other cell sources (which includes human corneal epithelium stem cells, peripheral arterial-derived stem cells, and induced pluripotent stem cell lines). In 2020, adipose tissue-derived MSCs accounted for the markets largest share due to their increasing utilization in treating inflammatory diseases and wounds & injuries. There are several associated advantages, such as ease of harvesting stem cells by minimally invasive methods, simplicity of the isolation procedure, and better quality & proliferation capacity of adipose tissue-derived stem cells.
The musculoskeletal disorders segment accounted for the largest share of the therapeutic application segment in the Stem Cell Therapy Market in 2020
Based on therapeutic application, the global market is segmented into musculoskeletal disorders, wounds & injuries, cardiovascular diseases, surgeries, inflammatory & autoimmune diseases, neurological disorders, and other therapeutic applications (which include ocular diseases, fat loss, and peripheral arterial diseases). In 2020, the musculoskeletal disorders segment accounted for the largest share of the therapeutic application segment. The large market share of this segment is attributed to the increasing prevalence of musculoskeletal disorders such as osteoarthritis, bone repair, and regeneration
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The Asia Pacific region is the fastest-growing region of the Stem Cell Therapy Market in 2020.
The Asia Pacific region is estimated to grow at the highest CAGR in the market during the forecast period. Some of the major factors fueling the growth of the APAC market include regulatory approvals and guidelines for product approvals and the presence of major stem cell players in countries such as South Korea, Japan, India, and Australia.
Key players in the Stem Cell Therapy Market include Smith & Nephew (UK), MEDIPOST Co., Ltd. (South Korea), Anterogen Co., Ltd. (South Korea), PHARMICELL Co., Ltd. (South Korea), JCR Pharmaceuticals Co., Ltd. (Japan), and NuVasive, Inc. (US).
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Two local sisters share an unbreakable bond after bone marrow donation – Riverhead News Review – Riverhead News Review
By daniellenierenberg
Lorraine LaRosa faced a seemingly impossible decision.
She knew how fortunate she was to have not one, but three perfect matches for a bone marrow transplant, a procedure used to treat several cancerous and noncancerous diseases, such as leukemia and Hodgkins lymphoma. The statistics for finding a perfect match can be grim. The best odds rest in an immediate family member. Otherwise, a patient must rely on the bone marrow registry and the slim chance of matching with a stranger.
Ms. LaRosa had the benefit of a large family. Out of seven siblings, four were healthy enough to be tested. And three came back as matches: her sisters Jennifer Lappe and Melissa Senatore, who live in Calverton, and her brother Jason Klinge of Southampton.
I was in tears because I didnt know what to do and who was the better choice, Ms. LaRosa said.
A sense of urgency had arrived in early 2020. Ms. LaRosa, 62, who lives in Mattituck, was undergoing frequent blood transfusions due to a lack of platelets and low red blood cells.
Things were getting pretty bad at that point, she said.
She didnt want to put the burden on any of her siblings. She called her doctor to talk through her concerns. The time had come to move forward, so the doctor took the decision out of her hands.
After careful evaluation of all three siblings health and medical histories and considering Ms. LaRosas worsening situation the doctors choice became clear. The donor would be her sister Jennifer.
Ms. Lappe understood her sisters hesitancy to ask forsuch a serious commitment. But there was never a moment of doubt. She had seen her sister struggle for years with her illness and have to endure the uncertainty of misdiagnosis and multiple procedures.
I knew she was scared, Ms. Lappe said. Id be scared with what she had to go through. But shes a lot stronger than I think she thinks she is.
Ms. LaRosa texted her sister with the news that unfortunately she would be the donor.
To me there was never a question, said Ms. Lappe, 60. Ill do whatever you need. Im in a million percent. I said, Im selfish, I love you. I want you to be around forever.
The sisters, who were always so close from a young age and grew up in a tight-knit family, would soon form an unbreakable bond one saving the others life.
Before Ms. LaRosa received an ultimate diagnosis of myelodysplastic syndrome, or MDS, in February 2020, she endured years of joint pain and symptoms that seemed to mystify her doctors. One time when her shoulder hurt, she was told it was a torn rotator cuff, which turned out to be inaccurate. Before that, when she was struggling to walk with swollen feet, a podiatrist said she had a torn Achilles tendon, but she hadnt done anything that seemed to warrant an injury typically seen in athletes. Lupus was also incorrectly diagnosed.
She struggled on a continual journey from one doctor to another.
She ended up in an emergency room on Feb. 27, 2017, and a doctor there noticed that her platelets the smallest blood cells seemed low.
Two months later, a doctor at New York Cancer & Blood Specialists diagnosed large granular leukemia, a rare cancer of white blood cells. As time went on, however, her condition did not improve.
Ms. LaRosas daughter, Taylor, described her mom as a fighter who was always optimistic and never overly concerned about her health issues.
I was more so the worrywart, said Taylor, 29. I kind of forced her to go to all these appointments and all of these doctors chasing all of these kind of vague symptoms. No one could kind of come up with what was going on.
A 2009 Mattituck High School graduate, Taylor works as a physician assistant at Weill Cornell Medical Center in New York City. She connected her mom with Dr. Gail Roboz, who specializes in hematology/oncology, in November 2017. Dr. Roboz became well known as the doctor for Robin Roberts, an anchor of ABCs Good Morning America, who was diagnosed with MDS in 2012.
[Dr. Roboz] kind of took on my case and was monitoring me and was saying my blood didnt really make any sense, Ms. LaRosa said. There were mutations in my blood that werent making sense for the large granular leukemia diagnosis.
Extensive testing revealed that Ms. LaRosa had a predisposition to MDS, a bone barrow disorder and blood cancer that often goes unrecognized and under-diagnosed, according to the MDS Foundation.
Then it was kind of a weird watching and waiting game, Taylor said. I think we all hoped it couldnt turn into this [MDS], but we knew it could.
Low-risk patients who do not receive a bone marrow transplant face an average survival rate of up to six years, according to the MDS Foundation. High-risk patients face as little as five months.
Taylor said she braced herself for the possibility that her mother would need a bone marrow transplant at some point. Each December, her mother would undergo a checkup with her oncologist.
Taylor examined her mothers lab work after the December 2019 appointment and could see the results were abnormal. The family had booked a cruise around Christmastime, so Taylor reached out to her mothers oncologist to see if it would be safe to travel.
Dr. Roboz gave them the OK and said theyd deal with it when they returned.
We went on this cruise and I didnt know anything, Ms. LaRosa said. My husband didnt know anything, but my daughter had all this information. She had some emotional moments on the cruise. Now, looking back, I know why.
Taylor recalled the trip to the Bahamas like something out of a movie, where nothing went wrong.
It was like a perfect trip, she said.
When Ms. LaRosa returned to her doctor for a follow-up, the reality of the situation set in. Dr. Roboz referred her to Dr. Tsiporah Shore, who has expertise in bone marrow transplants, at Weill Cornell Medicine. They met on March 14, 2020.
She basically said we need to do this right away, Ms. LaRosa said. Things were really progressing.
Ms. Lappe could see her sisters health was declining.
Nothing they did was making her better and I know this was something she dreaded doing, she said.
Before determining who would be the match, Ms. Lappe said she underwent the most extensive testing of her life. To find a match, doctors analyze the patients tissue type, specifically the human leukocyte antigen, or HLA, tissue, the proteins found on most cells in the body, according to the nonprofit organization Be the Match.
Finding the match is just the initial step in assuring that the donor is suitable for the transplant and there are no other potential ailments that could be passed on.
Ms. Lappe had assumed her brother Jason would be the pick since she had an autoimmune disease and he did not. When she found out she had been selected, it coincided with the early stage of the pandemic. That added another layer of stress, since Ms. Lappe knew if she came down with the virus, it could upend the entire process.
Other questions loomed over her.
Youre worrying, is my body going to do what it needs to do? Is it going to work? Will her body reject it? she said.
To begin the donation last July, Ms. Lappe received injections to increase her white blood cell count. At the same time, her sister was undergoing radiation and chemotherapy to essentially wipe her immune system clear, eliminating a lifetime of protections that had been built up.
Ms. Lappe said she had been warned shed feel pain in her bones from the shots. When she didnt feel anything after the first shot, she worried it might not be working.
By the third and fourth shot, there was no mistaking the odd sensation.
You have these bone pains, she said. Ive never had that happen.
On the fifth day, the doctors did a blood test as the final determination to begin the donation process.
To read more about bone marrow donation, visit BeTheMatch.org.
The procedure, called peripheral blood stem cell donation, required Ms. Lappe to be connected to a machine for six hours as blood was removed via a port in her chest to separate out the blood-forming cells. The remaining blood circulated back into her body.
At the end of it, one bag of the pinkish liquid that would be used to save her sister had been accumulated.
I said to her afterwards, it was so emotional, Ms. Lappe said, adding that she knew she would feel an overwhelming sense of guilt if the procedure didnt work.
She took a picture of the bag and its label, which read, Donor: Jennifer Lappe and Recipient: Lorraine LaRosa. She texted the picture to her sister and said, Oh, my gosh.
Ms. Lappe finished her donation on a Wednesday and her sister began to receive her bone marrow the next morning, once the doctors had determined they had a sufficient number of stem cells to start the process.
Then the waiting game began.
The day of a transplant is Day Zero. Every day afterward continues an upward count toward engraftment, when the blood-forming cells received during the transplant begin to grow and create healthy blood cells.
I would say those days were the hardest, just waiting, Taylor said. They would draw her labs every morning at 4 a.m. and the results would be back at 6 a.m.
A nurse would write the number on a board, and for several days it remained at zero. To pass the time, Ms. LaRosa would play games like Yahtzee with her husband, Mark, who commuted each day into the city. Taylor would watch Netflix shows like Jane the Virgin with her mom. The days were largely a blur for Ms. LaRosa.
Taylor knew it could take one to two weeks for engraftment to begin. It was Day 11 when they saw the first sign of hope as a nurse wrote .1 on the board, signaling the first sign of growth.
I remember that day being like a huge relief and huge turn, Taylor said.
Ms. LaRosa spent over a month in the hospital for close monitoring as her counts continued to climb. Even after she was released, she had to stay at a nearby hotel for another week because of daily checkups. She set her sights on Day 100, another milestone moment in the recovery.
If you make past Day 100, its a good thing, she said.
Even after a successful procedure with a 100% match, theres never a moment of being entirely in the clear. Ms. LaRosa will continue to be monitored for the rest of her life and setbacks are always possible. Shes faced one setback already, with graft vs. host disease, which can be common after a bone marrow transplant. Shes also endured blood clots.
But the biggest thing is that shes now clear of MDS and feeling better than before the procedure. She still, however, faces residual effects from chemotherapy. Shes often tired.
When she returned home, she mostly stayed inside, unable to venture out with the threat of COVID-19 still hanging over everything. Her immune system was rebuilding from scratch. She remains on a special diet. She cant have plants in the house, which put her at risk of exposure to pathogens that can cause disease. She cant have alcohol.
I said, God, I really want a glass of wine, Ms. LaRosa said with a laugh.
Taylor said there are constant signs of progress. Her mother just recently had a port removed from her chest wall after close to nine months. She received her COVID-19 vaccine. Her hair is growing back.
Shes starting to like the style, Taylor said.
She looks forward to the next steps of returning to normal: going to a movie theater and eating dinner at their favorite restaurant, Grana in Jamesport. When they sit together and toast their wine glasses, Taylor said she knows shell cry. They have always shared a close bond, particularly since Taylor was adopted at around 3 months old after her mother endured years of infertility issues.
Shes been my best friend and my rock for my whole life, Taylor said.
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Two local sisters share an unbreakable bond after bone marrow donation - Riverhead News Review - Riverhead News Review
Bone Marrow Processing Systems Market Quantitative Market Analysis, Current and Future Trends – Good News Gum
By daniellenierenberg
Bone marrow aspiration and trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.
The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.
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Europe and North America spearheaded the market as of 2018, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.
In 2018, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.
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Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy. Japan and China are the biggest markets for harvesting systems in Asia Pacific. Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.
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Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others.
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Bone Marrow Processing Systems Market Quantitative Market Analysis, Current and Future Trends - Good News Gum
Desperate family of boy, 9, with leukaemia have 10 days to save his life… – The Sun
By daniellenierenberg
THE FAMILY of a nine-year-old leukaemia patient have been given 10 days to raise funds for life-saving treatment.
Nathaniel Nabenas family are appealing as he "clings on to life" after they were told they have until May 12 to find 201,000 for a stem cell transplant.
2
Without the operation, his cancer will be terminal.
Nathaniel is not entitled to free NHS treatment because he is not a British national.
He flew to the UK to have a 5,000 prosthetic eye fitted privately after losing it to a tumour in his home country Nigeria.
Doctors at Londons Great Ormond Street Hospital, moved by Nathaniels plight, have revealed they have waived their private consultant fees to help.
And Paul OGrady, who presents ITV series Little Heroes at the childrens hospital, has voiced his support.
It was only when he arrived here in November that doctors discovered he had acute myeloid leukaemia, a cancer so aggressive that he could have died within weeks without chemotherapy.
A stem cell match has been found but the family now have to find 201,103.
This money goes to the NHS for the cost of the transplant, treatment and after-care, based on a typical in-patient admission of eight weeks and a three-month follow-up as an outpatient.
2
Nathaniels cancer is in remission after six rounds of chemo but his consultant says it could return at any time.
If they raise enough, then a transplant will go ahead after tests due to take place on May 14.
Parents Ebisidor, a business analyst, and wife Modupe, 38, who are staying with family in Croydon, South London, were initially told the hospital bill could be as much as 825,000.
Ebisidor, 45, told the Mirror: Weve seen a dramatic turnaround from the hopeless situation we were in six months ago and we cant thank Sunday People readers enough.
Its incredible that the doctors are treating him in their private work without charging. They are wonderful. We are so grateful to everyone for giving us hope but at the same time asking people to help Nathaniel cling on to life. We know its a lot to ask.
Professor Ajay Vora, a consultant paediatric haematologist at GOSH, said the superhero fan had been incredibly brave.
But he warned: The cancer could come back at any time and the longer we wait the more likely it will return. Then Nathaniel will only have the option of palliative care.
"The tests we are doing in two weeks will reassure us it hasnt started to come back before we give him the transplant.
Prof Vora added: All the consultants involved in his care are working in a private capacity and have waived their fee because they want to help him.
Our time is not borrowed from the NHS because we are treating Nathaniel in our private service in our time.
Doctors had hoped Nathaniel would be able to have a bone marrow transplant from one of his two sisters Nadia, 11, and Nicole, 21 months. But they were not a match.
Instead, stem cells from a stored umbilical cord will be used to save him.
Doctors will give Nathaniel high doses of chemotherapy to kill off his stem cells and replace them with healthy ones.
Dad Ebisidor said: The faster we can do this transplant the more chance Nathaniel has of survival.
"We dont have this sort of treatment back home. We didnt bring him to the UK sick. He got poorly while he was here. If the operation doesnt work our only option will be to take him to a hospice.
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By law, non-UK residents get free emergency care but are charged for operations if they are admitted to hospital.
They pay for treatment at 150% of the NHS national tariff the cost normally incurred for eligible patients.
A Great Ormond Street spokesman said: Nathaniel has responded well to treatment, with our clinical teams working to provide the best care for him including looking at taking advantage of the short window of time for receiving a transplant.
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Desperate family of boy, 9, with leukaemia have 10 days to save his life... - The Sun
Hematopoietic Stem Cell Transplantation (HSCT) Global Market Report (2020-2027) Segmented by Type, Application and region (NA, EU, and etc.) The…
By daniellenierenberg
The objective of the study is to define market sizes of different segments and countries in previous years and to forecast the values to the next Five years. The report is designed to incorporate both qualify qualitative and quantitative aspects of the industry with respect to each of the regions and countries involved in the study. Furthermore, the report also caters the detailed information about the crucial aspects such as drivers and restraining factors which will define the future growth of the Hematopoietic Stem Cell Transplantation (HSCT) market.
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Major Participators LandscapeThese market players enjoyed broad industry coverage, outstanding operational ability, and strong financial resources. Manufacturers are focusing on product innovation, brand extension, and the introduction of new brands to cater to the preferences of consumers. Some of them will be endowed with vital future while others will show a weak growth during the prospective timeframe.Major market participators covered in our report are:Cryo-Save AG ViaCord Inc Lonza Group Ltd Pluristem Therapeutics Inc China Cord Blood Corp CBR Systems Inc Regen Biopharma Inc Escape Therapeutics Inc
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Hematopoietic Stem Cell Transplantation (HSCT) Market: Application OutlookPeripheral Blood Stem Cells Transplant (PBSCT) Bone Marrow Transplant (BMT) Cord Blood Transplant (CBT)
By Type:Allogeneic Autologous
Table of Content1 Report Overview1.1 Product Definition and Scope1.2 PEST (Political, Economic, Social and Technological) Analysis of Hematopoietic Stem Cell Transplantation (HSCT) Market2 Market Trends and Competitive Landscape3 Segmentation of Hematopoietic Stem Cell Transplantation (HSCT) Market by Types4 Segmentation of Hematopoietic Stem Cell Transplantation (HSCT) Market by End-Users5 Market Analysis by Major Regions6 Product Commodity of Hematopoietic Stem Cell Transplantation (HSCT) Market in Major Countries7 North America Hematopoietic Stem Cell Transplantation (HSCT) Landscape Analysis8 Europe Hematopoietic Stem Cell Transplantation (HSCT) Landscape Analysis9 Asia Pacific Hematopoietic Stem Cell Transplantation (HSCT) Landscape Analysis10 Latin America, Middle East & Africa Hematopoietic Stem Cell Transplantation (HSCT) Landscape Analysis 11 Major Players Profile
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Regional Segment AnalysisThe report focuses on detailed analysis of major regions like North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India and Southeast Asia), South America (Brazil, Argentina, Columbia), and Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa).
Report Key AudienceHematopoietic Stem Cell Transplantation (HSCT) manufacturersDownstream vendors and end-usersTraders, distributors, and resellers of Hematopoietic Stem Cell Transplantation (HSCT)Hematopoietic Stem Cell Transplantation (HSCT) industry associations and research organizationsProduct managers, Hematopoietic Stem Cell Transplantation (HSCT) industry administrator, C-level executives of the industriesMarket Research and consulting firms
Hematopoietic Stem Cell Transplantation (HSCT) Report Provide:Potential opportunities and challenges analysis in Hematopoietic Stem Cell Transplantation (HSCT) market.Current and future market outlook in the developed and emerging regional markets.Detailed analysis of the segment that is expected to dominate the market.Regions that are expected to witness the fastest growth during the forecast period.Identify the latest developments, market shares, and strategies employed by the major market players.Comprehensive & in-depth research and after-sales warranty by Global Market Monitor.Analysis of Influences of COVID-19 to the present and future Hematopoietic Stem Cell Transplantation (HSCT) market and related industry.
About Global Market MonitorGlobal Market Monitor is a professional modern consulting company, engaged in three major business categories such as market research services, business advisory, technology consulting.We always maintain the win-win spirit, reliable quality and the vision of keeping pace with The Times, to help enterprises achieve revenue growth, cost reduction, and efficiency improvement, and significantly avoid operational risks, to achieve lean growth. Global Market Monitor has provided professional market research, investment consulting, and competitive intelligence services to thousands of organizations, including start-ups, government agencies, banks, research institutes, industry associations, consulting firms, and investment firms.ContactGlobal Market MonitorOne Pierrepont Plaza, 300 Cadman Plaza W, Brooklyn,NY 11201, USAName: Rebecca HallPhone: + 1 (347) 467 7721Email: info@globalmarketmonitor.comWeb Site: https://www.globalmarketmonitor.com
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Hematopoietic Stem Cell Transplantation (HSCT) Global Market Report (2020-2027) Segmented by Type, Application and region (NA, EU, and etc.) The...
Galapagos creates new subscription right plans
By Dr. Matthew Watson
Mechelen, Belgium; 30 April 2021, 22.01 CET; regulated information –– Galapagos NV (Euronext & NASDAQ: GLPG) announced today that its supervisory board created 2,736,250 subscription rights under new subscription right plans for the benefit of the personnel of the company and its subsidiaries.
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Galapagos creates new subscription right plans
Progenity Presents Key Verification Study Data for Preecludia™ Preeclampsia Rule-out Test at the 2021 American College of Obstetricians and…
By Dr. Matthew Watson
Data demonstrate strong sensitivity and negative predictive value (NPV) Data demonstrate strong sensitivity and negative predictive value (NPV)
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Progenity Presents Key Verification Study Data for Preecludia™ Preeclampsia Rule-out Test at the 2021 American College of Obstetricians and...
ObsEva SA presents posters at the ACOG Annual Clinical and Scientific Virtual Meeting April 30 – May 2, 2021
By Dr. Matthew Watson
GENEVA, Switzerland and BOSTON, MA – April 30, 2021 – ObsEva SA (NASDAQ: OBSV / SIX: OBSN) a biopharmaceutical company developing and commercializing novel therapies to improve women’s reproductive health, today announced the presentation of five e-Posters on linzagolix in the treatment of uterine fibroids at the American College of Obstetricians and Gynecologists (ACOG) Annual Clinical and Scientific Meeting (ACSM), which will take place virtually from April 30 to May 2, 2021.
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ObsEva SA presents posters at the ACOG Annual Clinical and Scientific Virtual Meeting April 30 - May 2, 2021
ECMOHO Announces Fourth Quarter Unaudited Financial Results and Fiscal Year 2020 Audited Financial Results
By Dr. Matthew Watson
SHANGHAI, China, April 30, 2021 (GLOBE NEWSWIRE) -- ECMOHO Limited (Nasdaq: MOHO) (“ECMOHO” or the “Company”), a leading integrated solutions provider in the non-medical health and wellness market in China, today announced its audited financial results for the fiscal year ended December 31, 2020.
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ECMOHO Announces Fourth Quarter Unaudited Financial Results and Fiscal Year 2020 Audited Financial Results
AnPac Bio Announces Filing of Annual Report on Form 20-F for 2020
By Dr. Matthew Watson
SAN JOSE, Calif., April 30, 2021 (GLOBE NEWSWIRE) -- AnPac Bio-Medical Science Co., Ltd. (NASDAQ: ANPC) (“AnPac Bio” or the “Company”), a biotechnology company with operations in China and the United States focused on early cancer screening and detection, announced today that it has filed its annual report on Form 20-F for the fiscal year ended December 31, 2020 with the U.S. Securities and Exchange Commission (the “SEC”) on April 30, 2021.
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AnPac Bio Announces Filing of Annual Report on Form 20-F for 2020
Blueberries Medical Reports 2020 Financial Results
By Dr. Matthew Watson
TORONTO, April 30, 2021 (GLOBE NEWSWIRE) -- Blueberries Medical Corp. (CSE: BBM) (OTC: BBRRF) (FRA: 1OA) the Canadian parent of Blueberries S.A.S. (“BBSAS”), the premier Latin American licensed cultivator and producer of medicinal cannabis and medicinal-grade cannabis extracts, (together the “Company” or “Blueberries”), is pleased to report its financial results for the year ended December 31, 2020. Blueberries has filed today its audited consolidated financial statements and related management’s discussion and analysis, both of which are available on Blueberries’ profile at www.sedar.com. All financial information in this press release is reported in Canadian dollars, unless otherwise indicated.
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Blueberries Medical Reports 2020 Financial Results
Gracell Biotechnologies to Participate in Upcoming Virtual Conferences in May
By Dr. Matthew Watson
SUZHOU and SHANGHAI, China, April 30, 2021 (GLOBE NEWSWIRE) -- Gracell Biotechnologies Inc. (NASDAQ: GRCL) (“Gracell”), a global clinical-stage biopharmaceutical company dedicated to discovering and developing highly efficacious and affordable cell therapies for the treatment of cancer, today announced that it will participate in the following upcoming virtual conferences:
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Gracell Biotechnologies to Participate in Upcoming Virtual Conferences in May
Shattuck Labs Announces Participation in Upcoming May Conferences
By Dr. Matthew Watson
AUSTIN, TX and DURHAM, NC, April 30, 2021 (GLOBE NEWSWIRE) -- Shattuck Labs, Inc. (Shattuck) (NASDAQ: STTK), a clinical-stage biotechnology company pioneering the development of bi-functional fusion proteins as a new class of biologic medicine for the treatment of patients with cancer and autoimmune disease, today announced that Taylor Schreiber, M.D., Ph.D., Shattuck’s Chief Executive Officer, will participate in two upcoming conferences.
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Shattuck Labs Announces Participation in Upcoming May Conferences
Eve & Co Announces Financial Results for the Year Ended December 31, 2020
By Dr. Matthew Watson
STRATHROY, Ontario, April 30, 2021 (GLOBE NEWSWIRE) -- Eve & Co Incorporated (the “Company” or “Eve & Co”) (TSX-V: EVE; OTCQX: EEVVF) is pleased to announce its financial results for the year ended December 31, 2020. The financial statements and management discussion and analysis for such period are available on the System for Electronic Document Analysis and Retrieval ("SEDAR") at www.sedar.com and on Eve & Co's website at www.evecannabis.ca.
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Eve & Co Announces Financial Results for the Year Ended December 31, 2020
Novartis Phase III Beovu® data show potential for fluid resolution in more diabetic macular edema patients with fewer injections versus aflibercept
By Dr. Matthew Watson
Basel, May 1, 2021 — Novartis today announced positive one-year results of the Phase III KESTREL and KITE* studies, evaluating the efficacy and safety of Beovu® (brolucizumab) 6 mg in diabetic macular edema (DME). Both studies met their primary endpoints of non-inferiority in change in best corrected visual acuity (BCVA) from baseline for Beovu 6 mg versus aflibercept 2 mg at year one1. In KESTREL, patients on Beovu 6 mg gained a mean of 9.2 letters versus 10.5 letters for patients on aflibercept 2 mg1. In KITE, patients on Beovu 6 mg gained a mean of 10.6 letters versus 9.4 letters for patients on aflibercept 2 mg1. These results will be presented at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting.
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Novartis Phase III Beovu® data show potential for fluid resolution in more diabetic macular edema patients with fewer injections versus aflibercept
Editas Medicine to Present Preclinical Data Demonstrating Advancements in In Vivo Gene Editing Approach for the Treatment of Genetic Ocular Diseases…
By Dr. Matthew Watson
Preclinical data support novel approaches for the treatment of Usher Syndrome 2A and retinitis pigmentosa 4 Preclinical data support novel approaches for the treatment of Usher Syndrome 2A and retinitis pigmentosa 4
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Editas Medicine to Present Preclinical Data Demonstrating Advancements in In Vivo Gene Editing Approach for the Treatment of Genetic Ocular Diseases...
Oyster Point Pharma Announces Clinical Data Presentation of OC-01 (varenicline) Nasal Spray for Dry Eye Disease at the Association for Research in…
By Dr. Matthew Watson
PRINCETON, N.J., May 01, 2021 (GLOBE NEWSWIRE) -- Oyster Point Pharma, Inc. (Nasdaq: OYST), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class therapies to treat ocular surface diseases, announced today the presentation of data analyses from its Phase 3 ONSET-2 clinical trial evaluating OC-01 (varenicline) nasal spray for the treatment of signs and symptoms of dry eye disease at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Virtual Annual Meeting, being held on May 1-5.
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Oyster Point Pharma Announces Clinical Data Presentation of OC-01 (varenicline) Nasal Spray for Dry Eye Disease at the Association for Research in...
Denali Therapeutics Presents Positive Results from Phase 1 and Phase 1b Studies of Its LRRK2 Inhibitor, BIIB122/DNL151, Supporting Late-Stage…
By Dr. Matthew Watson
SOUTH SAN FRANCISCO, Calif., May 01, 2021 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (NASDAQ: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for neurodegenerative diseases, today announced final results from Phase 1 and Phase 1b studies of its small molecule LRRK2 inhibitor, BIIB122/DNL151, which is being developed in collaboration with Biogen as a potential treatment of Parkinson’s disease. Safety and biomarker goals were met in both studies, supporting plans to advance BIIB122/DNL151 into late-stage clinical development in Parkinson’s disease by year-end 2021. The results will be presented at the International Association of Parkinsonism and Related Disorders Virtual Congress, being held May 1-4.
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Denali Therapeutics Presents Positive Results from Phase 1 and Phase 1b Studies of Its LRRK2 Inhibitor, BIIB122/DNL151, Supporting Late-Stage...