A LITTLE boy faces his third bone marrow transplant before his second birthday.

Jack Kleinberg has battled against two life-threatening conditions as he suffers from familial mediterranean fever and WiskottAldrich syndrome, which affects one in 10 million children and means he has to live in virtual isolation.

His parents, Rob and Vicki, live with the knowledge that any part of his body can stop working at any time from a simple fall or infection.

The couple, of St James Gardens, Westcliff, spend much of their time travelling to Great Ormond Street Hospital for Jack to receive treatment to keep him alive.

The family includes Robs children from a previous relationship, Oliver, 14 and Sophia, ten.

Vicki, 28, said: Its a 24/7 job, but we wouldnt change it for the world. Oliver and Sophia didnt see Jack for the first year because he was in hospital. Its become normal for them to come home and wash and change into sterile clothes before they can see Jack, because of the danger of infection for him. Jack has had one full transplant and a top-up transplant and is waiting for a potential donor for a possible third transplant.

Vicki said: People think it is a painful process, but these days it is a stem cell transplant where if a donor is found to be suitable, they are given an injection the week before, which makes the body release bone marrowcells into the blood stream which are then taken like a normal blood donation. It takes just 20 minutes of someones time and saves so many lives. The transplants have given Jack 25 per cent of the cells he needs. Without them, he wouldnt have lived past his first birthday.

We are trying to get through Christmas and then we will decide on whether, if a donor is found, Jack has another full stem cell transplant or whether we let him live his life, with all its restrictions, for a while because he has spent so much time in hospital.

For more information about becoming a donor, visit http://www.anthonynolan.org

Rugby club is pitching in

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People urged to donate bone marrow as tot faces third transplant

Freeport, Grand Bahama (PRWEB) December 18, 2014

Okyanos, the leader in cell therapy, announced the expansion of its medical team to accommodate the growing demand for cell therapy to treat patients with chronic unmet needs for which adult stem cell therapy using cells from a persons own fat (adipose) tissue has been found to be safe and efficacious. Led by a prestigious team of U.S.-licensed physicians and nursing staff, the team includes Dr. Todd Malan, Chief Cell Therapy Officer and pioneer of adipose-derived stem cell therapy, and is joined by Dr. Matthew Mick, Cardiologist, FACC, Fellowship at Cleveland Clinic.

We are very pleased to have such a competent and highly regarded aggregate of expertise, said Okyanos CEO Matt Feshbach. Our team is comprised of leaders in their respective fields, each of whom is committed to bringing about a new standard of care and better quality of life to our patients.

Todd Malan, MD, serves as the Chief Cell Therapy Officer and General Surgeon at Okyanos, overseeing the fat-harvesting and stem cell isolation step of the Okyanos cell therapy process. A pioneer of fat-derived stem cell therapies, he became the first physician in the U.S. to utilize stem cells from fat for soft tissue reconstruction in October 2009, combining water-assisted fat-harvesting, fat transfer and adult stem cell technologies.

Matthew J. Mick, MD, is a triple board-certified interventional cardiologist. After attending the Indiana University School of Medicine, Dr. Mick completed his Cardiovascular Disease and Interventional Fellowships at the Cleveland Clinic Foundation. Dr. Mick participated as Principal Investigator and Co-Investigator in more than 20 cardiac clinical trials. He was a leader in developing trans-radial cardiac catheterization and holds several patents for cardiac catheters. Dr. Mick has performed over 15,000 diagnostic procedures in his 22 years of practice.

As the Director of Nursing managing a medical team which now numbers 10, Gretchen Dezelick oversees all of the clinical operations and maintains the superior cleanliness and safety standards that help make Okyanos a center of excellence. With more than 25 years of nursing experience progressing from bedside nursing to administrative and management positions in a variety of healthcare settings, Gretchen was a Certified Critical Care Nurse (CCRN) for more than 20 years and has been a Certified Peri-Operative Nurse (CNOR) for more than three years as well as being a Licensed Health Care Risk Manager (LHCRM).

Okyanos is also very proud to include several Bahamian medical staff such as Anesthesiologist Dr. Vincent Burton, Fellow of the Royal College of Anaesthetists, UK (FRCA), a Certified Critical Care Nurse, cardiology tech, sonographer, surgical scrub tech and a facilities tech, to deliver well-rounded expert patient care. The team also includes a Certified Cardiovascular Nurse, a BSN RN and a cardiovascular tech, providing more than 88 years of combined experience.

Okyanos follows the treatment guidelines laid out in clinical trials such as PRECISE and others which have demonstrated positive results from adult stem cell therapy. Okyanos cell therapy is performed in their newly constructed surgery center built to U.S. surgical standards and which also includes a state-of-the-art Phillips cath lab.

Adult stem cell therapy has emerged as a new treatment alternative for those who are restricted in activities they can no longer do but are determined to live a more normal life. Okyanos cell therapy uses a unique blend of adult stem cells derived from a patients own fat tissue, thereby helping the bodys own natural biology to heal itself.

Just 50 miles from US shore, Okyanos cell therapy is available to patients with severe heart disease including coronary artery disease (CAD) and congestive heart failure (CHF) as well as patients with autoimmune diseases, tissue ischemia, neurological and orthopedic conditions.

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Okyanos Expands World-Class Cell Therapy Medical Team

As the main component of connective tissue in the body, fibroblasts are the most common type of cell. Taking advantage of that ready availability, scientists from the Perelman School of Medicine at the University of Pennsylvania, the Wistar Institute, Boston University School of Medicine, and New Jersey Institute of Technology have discovered a way to repurpose fibroblasts into functional melanocytes, the body's pigment-producing cells. The technique has immediate and important implications for developing new cell-based treatments for skin diseases such as vitiligo, as well as new screening strategies for melanoma. The work was published this week in Nature Communications.

The new technique cuts out a cellular middleman. Study senior author Xiaowei "George" Xu, MD, PhD, an associate professor of Pathology and Laboratory Medicine, explains, "Through direct reprogramming, we do not have to go through the pluripotent stem cell stage, but directly convert fibroblasts to melanocytes. So these cells do not have tumorigenicity."

Changing a cell from one type to another is hardly unusual. Nature does it all the time, most notably as cells divide and differentiate themselves into various types as an organism grows from an embryo into a fully-functional being. With stem cell therapies, medicine is learning how to tap into such cell specialization for new clinical treatments. But controlling and directing the process is challenging. It is difficult to identify the specific transcription factors needed to create a desired cell type. Also, the necessary process of first changing a cell into an induced pluripotent stem cell (iPSC) capable of differentiation, and then into the desired type, can inadvertently create tumors.

Xu and his colleagues began by conducting an extensive literature search to identify 10 specific cell transcription factors important for melanocyte development. They then performed a transcription factor screening assay and found three transcription factors out of those 10 that are required for melanocytes: SOX10, MITF, and PAX3, a combination dubbed SMP3.

"We did a huge amount of work," says Xu. "We eliminated all the combinations of the other transcription factors and found that these three are essential."

The researchers first tested the SMP3 combination in mouse embryonic fibroblasts, which then quickly displayed melanocytic markers. Their next step used a human-derived SMP3 combination in human fetal dermal cells, and again melanocytes (human-induced melanocytes, or hiMels) rapidly appeared. Further testing confirmed that these hiMels indeed functioned as normal melanocytes, not only in cell culture but also in whole animals, using a hair-patch assay, in which the hiMels generated melanin pigment. The hiMels proved to be functionally identical in every respect to normal melanocytes.

Xu and his colleagues anticipate using their new technique in the treatment of a wide variety of skin diseases, particularly those such as vitiligo for which cell-based therapies are the best and most efficient approach.

The method could also provide a new way to study melanoma. By generating melanocytes from the fibroblasts of melanoma patients, Xu explains, "we can screen not only to find why these patients easily develop melanoma, but possibly use their cells to screen for small compounds that can prevent melanoma from happening."

Perhaps most significantly, say the researchers, is the far greater number of fibroblasts available in the body for reprogramming compared to tissue-specific adult stem cells, which makes this new technique well-suited for other cell-based treatments.

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New technology directly reprograms skin fibroblasts for a new role

MITTEILUNG UEBERMITTELT VON BUSINESS WIRE. FUER DEN INHALT IST ALLEIN DAS BERICHTENDE UNTERNEHMEN VERANTWORTLICH.

PARIS --(BUSINESS WIRE)-- 13.10.2014 --

Stem cells hold great promise for treating a variety of human diseases and injuries. Basic and translational stem cell research is among the most competitive fields in the life sciences. We have co-organized the first Bridging Biomedical Worlds conference of our new series of international scientific meetings: Turning Obstacles into Opportunities for Stem Cell Therapy.

The goal of this conference is to promote progress in the translation of basic stem cell research into stem cell therapies. To do this, presentations will highlight diverse areas of on-going stem cell biology research. In addition, panelists will discuss obstacles to translation and the associated risks and ethical controversies. These panels will provide a means to accelerate communication and cooperation among researchers, bioengineers, clinicians and industry scientists, and will explore ways to implement international policies, regulations and guidelines to ensure the development of safe and effective stem cell therapies worldwide. Participants will hear about the latest basic and translational stem cell research from more than 20 distinguished speakers from China, Japan, Europe and theUnited States.

This conference held in Beijing, China, October 13-15, 2014 is co-organized by the Fondation IPSEN, AAAS/Science and AAAS/Science Translational Medicine, in association withFred Gage (Salk Institute for Biological Studies) and Qi Zhou (Institute of Zoology, Chinese Academy of Sciences).

About AAAS/Science The American Association for the Advancement of Science (AAAS) is the worlds largest general scientific society and publisher of the journal Science (www.sciencemag.org) as well as Science Translational Medicine (www.sciencetranslationalmedicine.org) and Science Signaling (www.sciencesignaling.org). AAAS was founded in 1848, and includes some 261 affiliated societies and academies of science, serving 10 million individuals.Sciencehas the largest paid circulation of any peer-reviewed general science journal in the world, with an estimated total readership of 1 million. The non-profit AAAS (www.aaas.org) is open to all and fulfills its mission to advance science and serve society not only by publishing the very best scientific research but also through initiatives in science policy, international programs and science education. http://www.sciencemag.org

About AAAS/Science Translational Medicine Science Translational Medicine, launched in October 2009, is the newest journal published by AAAS/Science. The goal of Science Translational Medicineis to promote human health by providing a forum for communicating the latest biomedical research findings from basic, translational, and clinical researchers from all established and emerging disciplines relevant to medicine. Despite 50 years of advances in our fundamental understanding of human biology and the emergence of powerful new technologies, the translation of this knowledge into effective new treatments and health measures has been slow. This paradox illustrates the daunting complexity of the challenges faced by translational researchers as they apply the basic discoveries and experimental approaches of modern science to the alleviation of human suffering. A major goal ofScience Translational Medicineis to publish papers that identify and fill the scientific knowledge gaps at the junction of basic research and medical application in order to accelerate the translation of scientific knowledge into new methods for preventing, diagnosing and treating human disease. http://www.sciencetranslationalmedicine.org

About the Institute of Zoology, Chinese Academy of Sciences Institute of Zoology (IOZ), Chinese Academy of Sciences (CAS), is one of the leading research institutions in China. The institute consists of 76 professors (including 2 members of Chinese Academy of Sciences), 3 state key research laboratories and 1 zoological museum. The major research areas of IOZ include animal sciences, cell membrane biology, stem cells and reproduction. The stem cell research teams of IOZ include over 10 PIs, and they mainly focus on questions related to the establishment of pluripotent stem cell lines, neural stem cell induction and regeneration, mechanism studies of pluripotency and differentiation regulation of embryonic stem cells, animal model establishment and functional studies, etc. The major achievements in the field of stem cell research made by IOZ faculties include: obtained the first healthy animal (Xiaoxiao the mouse) using induced pluripotent stem cells (iPSCs) via tetraploid complementation method, identified molecular markers for the evaluation of pluripotency levels of stem cells and the related regulatory mechanisms, achieved cell fate conversion across different germ layers, established various types of human and mouse embryonic stem cell lines, as well as the Beijing Stem Cell Bank, etc. These achievements has once been selected as one of the TIMES Top 10 Medical Breakthroughs in 2009, and twice been selected as Top 10 Breakthroughs in Science and Technology in China. The Beijing Stem Cell Bank now functions as a resource for stem cell and regenerative medicine studies, providing various types of embryonic stem cell lines, adult stem cell lines and somatic cell lines for many research groups. IOZ also hosts modern animal model research centers for pigs and monkeys, which have generated a few valuable animal models for disease mechanism studies and pharmaceutical researches. http://www.english.ioz.cas.cn

About the Fondation Ipsen Established in 1983 under the aegis of the Fondation de France, the mission of the Fondation Ipsen is to contribute to the development and dissemination of scientific knowledge. The long-standing action of the Fondation Ipsen aims at fostering the interaction between researchers and clinical practitioners, which is essential due to the extreme specialization of these professions. The ambition of the Fondation Ipsen is to initiate a reflection about the major scientific issues of the forthcoming years. It has developed an important international network of scientific experts who meet regularly at meetings known as Colloques Mdecine et Recherche, dedicated to five main themes: Alzheimer's disease, neurosciences, longevity, endocrinology and cancer science. Moreover the Fondation Ipsen has started since 2007 several meetings in partnership with the Salk Institute, the Karolinska Institutet, the Massachusetts General Hospital, the Days of Molecular Medicine Global Foundation as well as with the science journals Nature, Cell and Science. The Fondation Ipsen has published over one hundred books and has awarded more than 250 prizes and research grants. http://www.fondation-ipsen.org

Fondation Ipsen For further information, please contact: Isabelle de Segonzac, Image Sept E-mail : isegonzac@image7.fr Tel. : +33 (0)1 53 70 74 70

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BUSINESS WIRE: The 1st Meeting of the Series Bridging Biomedical Worlds: Turning Obstacles into Opportunities for ...

Scientists at UB and other institutions have turned cells normally used as model cells, known as immortalized cells, into stem or, as they call it, stem-like cells, using nothing more than mechanical stress. They have done it without employing the potentially hazardous techniques previously used to obtain similar results.

The researchers use the term stem-like cells to describe cells in tissue culture that have many of the biochemical markers of stem cells. Determining whether or not they can differentiate will be the focus of future research.

The finding is described in a paper published recently online before print in the Proceedings of the National Academy of Sciences. The researchers discovered that changing the mechanical stresses on neuronal and other cell types in tissue culture allowed them to be reprogrammed into stem-like cells.

Normal cell types in tissue culture are spread out and have differentiated internal structures, but changing cell mechanics caused the cells to turn into clusters of spherical cells that had many of the biochemical markers of cells, says Frederick Sachs, SUNY Distinguished Professor in the Department of Physiology and Biophysics and senior author.

The stem cell advance was made possible by the development of a genetically encoded optical probe by Fanje Meng, research assistant professor in the Department of Physiology and Biophysics and lead UB author. The probe measures the mechanical stress in actin, a major structural protein present in all cells. Actin is involved in muscle contraction and numerous cellular processes, including cell signaling, how cells are shaped and how they move.

The actin probes will provide researchers with a method of studying how mechanical forces influence living cells, tissues, organs and animals in real time.

This probe allows us, for the first time, to measure the stress in actin within living cells, explains Sachs. We saw gradients of stress in actin filaments even in single living cells.

Much of existing biomechanics will have to be rethought, since many studies have assumed that the stresses are uniform, Sachs continues. The actin stress probe showed that the tension in actin fibers in stem cells is higher than in normal cells. That was very surprising to us.

He adds that while mechanics are well known to have a role in cellular processes, the details are poorly understood because there have been few ways to measure the stress in specific proteins. A clinically relevant example is that metastatic cancer cells, the fatal variety, have different mechanics than cells of the parent tumor.

This probe will allow cancer researchers to better understand what allows cells to become metastatic, says Sachs.

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Mechanical cues reprogram normal cell lines into stem-like cells

Tommy #39;s Experience with Stem Cell Therapy
Tommy discusses living with debilitating back pain and choosing stem cell therapy followed by hyperbaric oxygen therapy to improve his quality of life. Learn more at http://beyondpills.com/,...

By: PainSpecialistCenter

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Tommy's Experience with Stem Cell Therapy - Video

Improved case of Anky Spondy after PRP and Stem Cell Therapy
stem cell india, stem cell therapy india, stem cell in india, stem cell therapy in india, india stem cell, india stem cell therapy.

By: Stem Cell India

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Improved case of Anky Spondy after PRP and Stem Cell Therapy - Video

Stem Cell Therapy | stem cells osteoarthritis independent
http://www.arthritistreatmentcenter.com On the flip side, another study refutes the study I commented on the other day regarding stem cells from osteoarthritis patients Chondrogenic Potential...

By: Nathan Wei

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Stem Cell Therapy | stem cells osteoarthritis independent - Video

Experience a New Dimension in Skin Care

The RG-Cell is the latest, breakthrough, anti-aging, skin care cosmaceutical to hit the market. It features a unique proprietary blend of stem cell activators programmed to protect your skin and visibly fight aging at the cellular level.

Scientists have shown that reactivating your dormant stem cells is the most effective process for skin rejuvenation and regeneration. This process stimulates fibroblast production of collagen, increasing skin firmness and elasticity, while reducing the appearance of fine lines and wrinkles for a smoother, silkier, vibrant and younger looking skin.

The Mayo Clinic defines Stem cells are the body's raw materials: They are cells from which all other cells with specialized functions are generated. Under the right conditions in the body or in a laboratory, stem cells divide to form more cells, called daughter cells. These daughter cells either become new stem cells (self-renewal) or become specialized cells (differentiation) with a more specific function, such as blood cells, brain cells, skin cells or heart muscle or bone. Stem cells are unique no other cell in the body has the natural ability to generate new cell types.

They can divide (through mitosis where they split into 2 separate but identical sets with 2 separate nuclei) or differentiate into diverse and specific cell types and can self-renew to produce more stem cells. In mammals, there are two broad types of stem cells:

Many specialized cells, such as in the skin, or blood, have a lifespan of only a few days. For these tissues to function, a steady replenishment of specialized cells is indispensable.

First, they are able to differentiate into all the different cell types that make up their respective tissue a property called pluripotency.

Second, they need to renew themselves in order to be able to supply new specialized tissue cells throughout life.

Skin is an essential tissue in our bodies. It is our bodys largest organ. Our skin protects us from infection, irritation and dehydration, and allows us to feel many different things, such as pressure, stress or heat. Our skin has to be constantly renewed throughout our lives and relies on a whole host of different stem cells to keep it in good shape.

Stem cells (SCs) residing in the epidermis and hair follicle ensure the maintenance of adult skin homeostasis and hair regeneration, but they also participate in the repair of the epidermis after injuries.

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Skin Care Treatment with RG-Cell Stem Cell Support Serum

At Stem Cell Treatment Institute advanced stem cell procedures are performed at some of the most scientifically advanced hospitals in the world. Stem cell therapy is focused on affecting physical changes in the Spinal Cord that can improve a patient's quality of life. Spinal Cord Injury patients can be treated by lumbar puncture (injecting the cells into the cerebrospinal fluid), IV, or other techniques. Typically this is an outpatient procedure. however patients may stay for 4 or 5 nights in our suites during the process.

Treatment using autologous (patient source) or donor cells (placenta) are available

If Autologous Bone Marrow is used bone marrow is collected from the patient's iliac crest (hip bone) using thin-needle puncture under local anesthesia. Once the bone marrow collection is complete, patients may return to their suite or hotel and go about normal activities.

The stem cells are then processed in a state-of-the-art laboratory. In the lab, both the quantity and quality of the stem cells are measured. The stem cells are then implanted back into the patient by lumbar puncture or IV.

Cost: Stem cell treatments begin around $13,500 (adults).

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We offer Stem Cell treatments with enhanced or manipulated stem cells. These expanded and mobilized stem cells have been found to provide better results than non-manipulated stem cell applications. Manipulation or amplification of the stem cells is done in the lab, where care is taken to retain the cell properties. These expanded and mobilized cells provide superior results and cell recovery has been found to occur twice as fast as with non-manipulated stem cell applications.

Studies where both types of cells were used show that results were quicker and were obtained predominantly from the manipulated stem cells.

Stem Cells can come from the patients fat or bone marrow, but stem cells from donor placenta or umbilical cord blood is also available and may have improved benefits. Donor characteristics (i.e., age) play a key role in treatment success. Your individual situation will be considered and suitable options will be discussed.

As we age our stem cells become less effective. For this reason younger cells are often preferred. We do not need to go all the way back to an early stage embryo to get young cells. Young cord blood cells can be used from The Placenta, Umbilical Cord, and other young sources. These cord blood cells are more likely than stem cells found in bone marrow to have proliferative properties. This means that stem cells found in cord blood have a greater ability to regenerate.

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Stem Cell Treatment for Spinal Cord Injury

Latest Hair Loss Research : Stem Cell Therapy and Stem Cell Nutrition for Hair Loss
For More Details Like Us : https://www.facebook.com/SuperStemCellNutrition.

By: Palu Sot

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Latest Hair Loss Research : Stem Cell Therapy and Stem Cell Nutrition for Hair Loss - Video

November 25, 2014

Chuck Bednar for redOrbit.com Your Universe Online

Two different teams of researchers, one led by scientists from The Scripps Research Institute (TSRI) and the other involving members of the Harvard Stem Cell Institute (HSCI) have discovered ways to create the neurons that detect pain, itch and other sensations in laboratory conditions out of human and mouse skin cells.

The TSRI study, which was published online Monday in the journal Nature Neuroscience, used what the authors referred to as a simple technique to create neurons that normally reside in clusters called dorsal root ganglia (DRG) along the outer spine. Those neurons are often affected by spinal cord injuries and a neurodegenerative condition known as Friedreichs ataxia.

According to the researchers, DRG sensory neurons extend their nerve fibers into skin, muscle and joints located throughout the body. The neurons are capable of alternately detecting gentle touch, painful contact, heat, cold, wounds, inflammation, chemical irritants, itch-inducing agents and fullness of the bowels and bladder. They also relay information about the position of the body and limbs, and have been linked to aging and autoimmune disease.

Due to the difficulties involved in culturing adult human neurons, most research relating to DRG neurons has been done in mice. However, the rodents are of limited use in understanding the human version of this somatosensory system, TSRI explained. The new discovery will allow this type of human neurons and their associated sensory mechanisms to be studied with relative ease in laboratory conditions, according to the study authors.

We have found a way to produce induced sensory neurons from humans where these genes can be expressed in their normal cellular environment, associate professor Kristin K. Baldwin, an investigator in TSRIs Dorris Neuroscience Center, said in a statement. This method is rapid, robust and scalable. Therefore we hope that these induced sensory neurons will allow our group and others to identify new compounds that block pain and itch and to better understand and treat neurodegenerative disease and spinal cord injury.

Similarly, the HSCI-led study, which included experts from Boston Childrens Hospital (BCH) and Harvards Department of Stem Cell and Regenerative Biology (HSCRB), was able to successfully convert mouse and human skin cells into pain-sensing neurons that responded to several different types of stimuli responsible for causing both acute and inflammatory pain.

The authors of this study, which also appeared in Wednesdays online edition of Nature Neuroscience, said that their research could help scientists better understand the different types of pain that we experience, as well as better identify why people respond to pain in different ways and why some individuals are more or less likely to develop chronic pain. It could also result in the development of improved pain-relieving medications.

The six-year project resulted in the creation of neuronal pain receptors that respond to both the types of intense stimuli triggered by a physical injury, and the more subtle stimuli triggered by inflammation which results in pain tenderness. The researchers report that the fact the neurons can respond to both the gross and fine forms of stimulation which produce separate types of pain in humans confirm that they are functionally normal.

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Pain-Sensing Neurons Created From Human, Mouse Skin Cells

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genucel - Intensive New Stem Cell Eye Therapy Treatment ...

Chuck Bednar for redOrbit.com Your Universe Online

A new stem cell gene therapy developed by researchers at UCLA is set to begin clinical trials early next year after the technique reportedly cured 18 children who were born without working immune systems due to a condition known as ADA-deficient Severe Combined Immunodeficiency (SCID) or Bubble Baby disease.

The treatment was developed by Dr. Donald Kohn, a member of the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, and his colleagues, and according to the university, it is able to identify and correct faulty genes by using the DNA of the youngsters born with this life-threatening condition.

Left untreated, ADA-deficient SCID is often fatal within the first year of a childs life, reports Peter M. Bracke for UCLA. However, after more than three decades of research, Dr. Kohns team managed to develop a gene therapy that can safely restore the immune systems of children with the disease by using their own cells and with no noticeable side effects.

All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy, instead they are all thriving with fully functioning immune systems, Dr. Kohn, who is also a professor of pediatrics and of microbiology, immunology and molecular genetics, said in a recent statement.

Children born with SCID have to be isolated in a controlled environment for their own safety, because without an immune system, they are extremely vulnerable to illnesses and infections that could be deadly. While there are other treatments for ADA-deficient SCID, Dr. Kohn noted that they are not always optimal or feasible for many children. The new technique, however, provides them with a cure, and the chance to live a full healthy life.

SCID is an inherited immunodeficiency that is typically diagnosed about six months after birth, the researchers said, and children with the condition are so vulnerable to infectious diseases that even the common cold could prove fatal to them. This particular form of the condition causes cells to not create ADA, an enzyme essential for the production of the white blood cells which are a vital component of a healthy, normally-functioning immune system.

Approximately 15 percent of all SCID patients are ADA-deficient, according to the university, and these youngsters are typically treated by being injected twice per week with the required enzyme. This is a process that must continue throughout a patients entire life, and even then it doesnt always work to bring their immune systems to optimal levels. Alternately, they could undergo bone marrow transplants from matched siblings, but those matches are rare and the transplanted cells themselves are often rejected by the childs body.

Dr. Kohn and his colleagues tested two therapy regimens on 18 ADA-deficient SCID over the course of two multi-year clinical trials starting in 2009. During the trials, the blood stem cells of the patients were removed from their bone marrow and genetically modified in order to correct the defect. All 18 of the patients were cured.

The technique used a virus delivery system first developed in Dr. Kohns laboratory in the 1990s a technique which inserts the corrected gene that produces the ADA into the blood forming stem cells in the bone marrow. The genetically corrected blood-forming stem cells will then produce the T-cells required to combat infections.

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New Stem Cell Treatment Found To Cure 'Bubble Baby' Disease

AVON LAKE, OH (WOIO) - When Shannon Goulding's bloodhound Butler tore a ligament in his knee his entire personality changed.

"He was sedentary, and he wasn't as active as before," said Goulding.

Dr. Petti a veterinarianat the Avon Lake Animal Clinic told Goulding, who also works at the clinic, suggested that stem cell therapy could help.

"Watching him walk he looked stiff and uncomfortable," said Petti.

The therapy was successful. Goulding said after four weeks after the surgery she could see a change the way Butler moved.

Stem cell therapy helps animals suffering from sore knees and joints by using their own fat cells.

"You take them from the patient, you process them, make them active, and then you re inject them into the parts of the animal that are giving them problems," said Petti.

Petti said Avon Lake Animal Clinic has helped about 15 animals with stem cell therapy and people from all over the country have been calling.

One injection of stem cells can last up to three years, and after that a second injection may be needed.

Stem cell therapy is also an expensive procedure. It ranges from $2,000-2,500, but for Goulding she says seeing Butler run free without pain is worth it.

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Local clinic treats animals with stem cell therapy

PUBLIC RELEASE DATE:

20-Nov-2014

Contact: Krista Conger kristac@stanford.edu 650-725-5371 Stanford University Medical Center @sumedicine

Mouse cells and tissues created through nuclear transfer can be rejected by the body because of a previously unknown immune response to the cell's mitochondria, according to a study in mice by researchers at the Stanford University School of Medicine and colleagues in Germany, England and at MIT.

The findings reveal a likely, but surmountable, hurdle if such therapies are ever used in humans, the researchers said.

Stem cell therapies hold vast potential for repairing organs and treating disease. The greatest hope rests on the potential of pluripotent stem cells, which can become nearly any kind of cell in the body. One method of creating pluripotent stem cells is called somatic cell nuclear transfer, and involves taking the nucleus of an adult cell and injecting it into an egg cell from which the nucleus has been removed.

The promise of the SCNT method is that the nucleus of a patient's skin cell, for example, could be used to create pluripotent cells that might be able to repair a part of that patient's body. "One attraction of SCNT has always been that the genetic identity of the new pluripotent cell would be the same as the patient's, since the transplanted nucleus carries the patient's DNA," said cardiothoracic surgeon Sonja Schrepfer, MD, PhD, a co-senior author of the study, which will be published online Nov. 20 in Cell Stem Cell.

"The hope has been that this would eliminate the problem of the patient's immune system attacking the pluripotent cells as foreign tissue, which is a problem with most organs and tissues when they are transplanted from one patient to another," added Schrepfer, who is a visiting scholar at Stanford's Cardiovascular Institute. She is also a Heisenberg Professor of the German Research Foundation at the University Heart Center in Hamburg, and at the German Center for Cardiovascular Research.

Possibility of rejection

A dozen years ago, when Irving Weissman, MD, professor of pathology and of developmental biology at Stanford, headed a National Academy of Sciences panel on stem cells, he raised the possibility that the immune system of a patient who received SCNT-derived cells might still react against the cells' mitochondria, which act as the energy factories for the cell and have their own DNA. This reaction could occur because cells created through SCNT contain mitochondria from the egg donor and not from the patient, and therefore could still look like foreign tissue to the recipient's immune system, said Weissman, the other co-senior author of the paper. Weissman is the Virginia and D.K. Ludwig Professor for Clinical Investigation in Cancer Research and the director of the Stanford Institute for Stem Cell Biology and Regenerative Medicine.

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Pluripotent cells created by nuclear transfer can prompt immune reaction, researchers find

Researchers at the Cedars-Sinai Heart Institute have found that injections of cardiac stem cells might help reverse heart damage caused by Duchenne muscular dystrophy, potentially resulting in a longer life expectancy for patients with the chronic muscle-wasting disease.

The study results were presented today at a Breaking Basic Science presentation during the American Heart Association Scientific Sessions in Chicago. After laboratory mice with Duchenne muscular dystrophy were infused with cardiac stem cells, the mice showed steady, marked improvement in heart function and increased exercise capacity.

Duchenne muscular dystrophy, which affects 1 in 3,600 boys, is a neuromuscular disease caused by a shortage of a protein called dystrophin, leading to progressive muscle weakness. Most Duchenne patients lose their ability to walk by age 12. Average life expectancy is about 25. The cause of death often is heart failure because the dystrophin deficiency leads to cardiomyopathy, a weakness of the heart muscle that makes the heart less able to pump blood and maintain a regular rhythm.

"Most research into treatments for Duchenne muscular dystrophy patients has focused on the skeletal muscle aspects of the disease, but more often than not, the cause of death has been the heart failure that affects Duchenne patients," said Eduardo Marbn, MD, PhD, director of the Cedars-Sinai Heart Institute and study leader. "Currently, there is no treatment to address the loss of functional heart muscle in these patients."

During the past five years, the Cedars-Sinai Heart Institute has become a world leader in studying the use of stem cells to regenerate heart muscle in patients who have had heart attacks. In 2009, Marbn and his team completed the world's first procedure in which a patient's own heart tissue was used to grow specialized heart stem cells. The specialized cells were then injected back into the patient's heart in an effort to repair and regrow healthy muscle in a heart that had been injured by a heart attack. Results, published in The Lancet in 2012, showed that one year after receiving the experimental stem cell treatment, heart attack patients demonstrated a significant reduction in the size of the scar left on the heart muscle.

Earlier this year, Heart Institute researchers began a new study, called ALLSTAR, in which heart attack patients are being infused with allogeneic stem cells, which are derived from donor-quality hearts.

Recently, the Heart Institute opened the nation's first Regenerative Medicine Clinic, designed to match heart and vascular disease patients with appropriate stem cell clinical trials being conducted at Cedars-Sinai and other institutions.

"We are committed to thoroughly investigating whether stem cells could repair heart damage caused by Duchenne muscular dystrophy," Marbn said.

In the study, 78 lab mice were injected with cardiac stem cells. Over the next three months, the lab mice demonstrated improved pumping ability and exercise capacity in addition to a reduction in heart inflammation. The researchers also discovered that the stem cells work indirectly, by secreting tiny fat droplets called exosomes. The exosomes, when purified and administered alone, reproduce the key benefits of the cardiac stem cells.

Marbn said the procedure could be ready for testing in human clinical studies as soon as next year. The process to grow cardiac-derived stem cells was developed by Marbn when he was on the faculty of Johns Hopkins University. Johns Hopkins has filed for a patent on that intellectual property and has licensed it to Capricor, a company in which Cedars-Sinai and Marbn have a financial interest. Capricor is providing funds for the ALLSTAR clinical trial at Cedars-Sinai.

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Cardiac stem cell therapy may heal heart damage caused by Duchenne muscular dystrophy

Sue Walters only chance of survival from leukaemiawas a stem cell transplant No one in her family matched her tissue type Doctors searched the worldwide donor register They found Nicola Gerber, a student from Mechern, near the French border

By Chloe Lambert for the Daily Mail

Published: 20:21 EST, 17 November 2014 | Updated: 04:28 EST, 18 November 2014

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When Sue Walters was diagnosed with leukaemia, she hoped that the best of medical science would be used to cure it.

What she could never have anticipated was that her life would be saved by an 18-year-old boy from a remote German village.

Sues only chance of survival was a stem cell transplant previously known as a bone marrow transplant.

What Nicola has done is amazing it really is a gift of life. If I hadnt had the transplant, it was unlikely Id have lived beyond three months,' said Sue Walters of her donor Nicola Gerber

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Donor: The German teenager who saved my life

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11 hours ago Neurons (green) are detected by TuJI whereas motoneurons are revealed in red by the visicular transporter of acetylcholine. Credit: Inserm/Martinat, Ccile

The motor neurons that innervate muscle fibres are essential for motor activity. Their degeneration in many diseases causes paralysis and often death among patients. Researchers at the Institute for Stem Cell Therapy and Exploration of Monogenic Diseases (I-Stem - Inserm/AFM/UEVE), in collaboration with CNRS and Paris Descartes University, have recently developed a new approach to better control the differentiation of human pluripotent stem cells, and thus produce different populations of motor neurons from these cells in only 14 days. This discovery, published in Nature Biotechnology, will make it possible to expand the production process for these neurons, leading to more rapid progress in understanding diseases of the motor system, such as infantile spinal amyotrophy or amyotrophic lateral sclerosis (ALS).

Human pluripotent stem cells have the ability to give rise to every cell in the body. To understand and control their potential for differentiation in vitro is to offer unprecedented opportunities for regenerative medicine and for advancing the study of physiopathological mechanisms and the quest for therapeutic strategies. However, the development and realisation of these clinical applications is often limited by the inability to obtain specialised cells such as motor neurons from human pluripotent stem cells in an efficient and targeted manner. This inefficiency is partly due to a poor understanding of the molecular mechanisms controlling the differentiation of these cells.

Inserm researchers at the Institute for Stem Cell Therapy and Exploration of Monogenic Diseases (I-Stem - Inserm/French Muscular Dystrophy Association [AFM]/University of vry Val d'Essonne [UEVE]), in collaboration with CNRS and Paris-Descartes University, have developed an innovative approach to study the differentiation of human stem cells and thus produce many types of cells in an optimal manner.

"The targeted differentiation of human pluripotent stem cells is often a long and rather inefficient process. This is the case when obtaining motor neurons, although these are affected in many diseases. Today, we obtain these neurons with our approach in only 14 days, nearly twice as fast as before, and with a homogeneity rarely achieved," explains Ccile Martinat, an Inserm Research Fellow at I-Stem.

To achieve this result, the researchers studied the interactions between some molecules that control embryonic development. These studies have made it possible to both better understand the mechanisms governing the generation of these neurons during development, and develop an optimal "recipe" for producing them efficiently and rapidly.

"We are now able to produce and hence study different populations of neurons affected to various degrees in diseases that cause the degeneration of motor neurons. We plan to study why some neurons are affected and why others are preserved," adds Stphane Nedelec, an Inserm researcher in Ccile Martinat's team.

In the medium term, the approach should contribute to the development of treatments for paralytic diseases such as infantile spinal muscular amyotrophy or amyotrophic lateral sclerosis. "Rapid access to large quantities of neurons will be useful for testing a significant number of pharmacological drugs in order to identify those capable of preventing the death of motor neurons," concludes Ccile Martinat.

Explore further: Team finds a better way to grow motor neurons from stem cells

More information: Combinatorial analysis of developmental cues efficiently converts human pluripotent stem cells into multiple neuronal subtypes, Nature Biotechnology, 17 Nov 2014. DOI: 10.1038/nbt.3049

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Production of human motor neurons from stem cells gaining speed