SPL

The bacterium Staphylococcus aureus is host to a smaller version of the enzyme used in the CRISPR technique for gene editing.

A tweak to a technique that edits DNA with pinpoint precision has boosted its ability to correct defective genes in people. Called CRISPR, the method is already used in the lab to insert and remove genome defects in animal embryos. But the genetic instructions for the machinery on which CRISPR relies a gene-editing enzyme called Cas9 and RNA molecules that guide it to its target are simply too large to be efficiently ferried into most of the human bodys cells.

This week, researchers report a possible way around that obstacle: a Cas9 enzyme that is encoded by a gene about three-quarters the size of the one currently used. The finding, published on 1April in Nature, could open the door to new treatments for a host of genetic maladies (F. A. Ran etal. Nature http://dx.doi.org/10.1038/nature14299; 2015).

There are thousands of diseases in humans associated with specific genetic changes, says David Liu, a chemical biologist at Harvard University in Cambridge, Massachusetts, who was not involved in the latest study. A fairly large fraction of those have the potential to be addressed using genome editing.

Genome editing has generated controversy, with unconfirmed reports of its use in human embryos. Some scientists have expressed concern that the technique might be used by fertility doctors to edit the genes of human embryos before its safety is established (see also E.Lanphier et al. Nature 519, 410411; 2015). That concern is exacerbated by the fact that changes made by the procedure in embryos would be passed to all subsequent generations without giving anyone affected the opportunity to consent (see Nature 519, 272; 2015). But in the non-reproductive cells of children and adults, where intergenerational issues are not a concern, researchers and companies are already racing to develop CRISPR as a clinical tool.

The ethics of that pursuit may be more straightforward, but its execution can be harder than using CRISPR in embryos. An embryo consists of a small number of cells that give rise to a human. To edit the genome at that stage is simply a matter of injecting the necessary CRISPR components into a few cells. An adult human, however, is a mix of trillions of cells assembled into many different tissues. Researchers fret over how to target the CRISPR machinery to the specific cells where defective genes are disrupting physiological processes.

You can have the most optimal gene-editing system in the world, but if you cant deliver it to the proper cell type, its irrelevant, says Nessan Bermingham, chief executive of Intellia Therapeutics in Cambridge, Massachusetts, which aims to bring genome editing to the clinic. Were spending a tremendous amount of time working on it.

Gene-therapy researchers often harness a virus called AAV to shuttle foreign genes into mature human cells. However, most laboratories use a gene encoding the Cas9 protein that is too large to fit in the snug confines of the AAV genome alongside the extra sequences necessary for Cas9 function.

Feng Zhang of the Broad Institute of MIT and Harvard in Cambridge, Massachusetts, and his colleagues decided to raid bacterial genomes for a solution, because the CRISPR system is derived from a process that bacteria use to snip unwanted DNA sequences out of their genomes. Zhangs team analysed genes encoding more than 600 Cas9 enzymes from hundreds of bacteria in search of a smaller version that could be packaged in AAV and delivered to mature cells.

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Mini enzyme moves gene editing closer to the clinic

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Pharmacogenomics: Personalized Medicine in Private Practice
Dr. Tommy Cawthon, M.D., Cardiology, discusses the benefits of a personalized approach to patient medication management as he has experienced in his practice.

By: GenoPATH, LLC

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Pharmacogenomics: Personalized Medicine in Private Practice - Video

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The latest in spinal cord research March 2, 2015 - Post session Q and A
Post presentation Q and A answered by a panel of SCI experts: Dr. Wolfram Tetzlaff, Dr. Lyn Jakeman Dr. Brian Kwon, Dr. Andrei Krassioukov, Dr. Chris McBride,

By: Spinal Cord Injury BC

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The latest in spinal cord research March 2, 2015 - Post session Q and A - Video

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The latest in spinal cord research March 2, 2015 - Dr. Christopher West
Dr. Christopher West talks about epidural stimulation on individuals with spinal cord injuries.

By: Spinal Cord Injury BC

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Stem Cell Transplant for Spinal Cord Injury | Quick Look
Spinal Cord Injury patient improves after Stem Cell Transplant such as his stamina, confidence, sitting balance and standing balance. Stem Cell Transplant done at Dr Alok Sharma NeuroGen Brain...

By: Neurogen Brain and Spine Institute

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Stem Cell Transplant for Spinal Cord Injury | Quick Look - Video

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Genesis Spinal Cord Injury Rehab Helps Russell Walk Again
After getting into a horrible car accident last July, Russell Evans never thought he #39;d walk again. But with the help of the Genesis Spinal Cord Injury Rehab program, Russell accomplished the...

By: Genesis Health System

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Golf Icon seeks new Regenerative Medicine treatment in Toledo
It sounds like something from a science fiction novel, but doctors say they can now use a patient #39;s own body to heal itself when it comes to joint damage. The practice is called Regenerative...

By: ProMedica

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No. 907 - Pharmakustik - Regenerative Medicine (Material Object Remix)
No. 907 - Pharmakustik - Tissue Engineering Track 6 - Regenerative Medicine (Material Object Remix) Released March 2015 https://no-ware.bandcamp.com/album/tissue-engineering http://no-wa.re.

By: No.

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Multiple sclerosis (MS) is a scary and unpredictable disease.

A patient's own immune system attacks the nervous system; causing numbness, dizziness and in some cases paralysis.

"It was terrifying, said Rachel Taylor. It was like having a wet, heavy blanket put over your life."

Taylor was diagnosed with MS 14 years ago.

"I was an active runner, and over the course of a few months couldn't figure out why I couldn't keep up," said Rachel.

Rachel knew what was wrong; she'd been working with the MS Society bike rides for years.

Rachel's in remission now, but she is still thrilled with Prof. Tom Lane's stem cell discovery.

"We have animals that are paralyzed that cannot right themselves, and once we en-graft the neural stem cells into the spinal cords, within three weeks, the majority of the animals, about 80 to 85 percent, will regain motor skills," said Prof. Tom Lane, PhD, a professor of Pathology at the University of Utah.

Researchers say MS damages the myelin, a layer around nerve cells.

Once injected, the human neural stem cells stimulate the mouse's own cells to repair the damage. When nerve cell function returns, the mice can walk and run again.

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Multiple sclerosis can be scary, but a new treatment holds promise

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Pharmacogenomics at Mayo Clinic with MedxPrime with Lee
Pharmacogenomics DNA testing is the future, or I should say the "now" of personalized medicine. reach out to me below... If you or someone you know takes, or will be taking medicine and you...

By: Lee Bane

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Newport Beach, California (PRWEB) March 31, 2015

Newport Beach based charity Coalition Duchenne has launched an interview series titled Making a Difference in Duchenne on its Youtube channel (https://www.youtube.com/user/CoalitionDuchenne) focused on individuals making a difference in Duchenne muscular dystrophy research, care, awareness, and education.

The first interview features Dr. Eduardo Marbn MD, PhD, director of the Cedars-Sinai Heart Institute in Los Angeles, talking about cardiac derived stem cells. Dr. Marbn was featured in a November 2011 Economist article Repairing Broken Hearts, read by Coalition Duchenne founder and executive director Catherine Jayasuriya. She lobbied for a focus on Duchenne because cardiac scarring severely compromises the life span of those with the disease. Coalition Duchenne funded successful research applying Marbns stem cell technology to Duchenne. The approach has been clinically proven to mitigate scarring cause by heart attacks. In Marbns therapy, human heart tissue is used to grow specialized heart stem cells, which are injected back into the patients heart.

We need to focus on changing the course of the disease. We lose many young men to cardiac issues. We hope that working with cardiac stem cells is one way we will eventually change that outcome, said Jayasuriya.

The second interview in the Making a Difference in Duchenne series features actor Cody Saintgnue, who plays Brett Talbot in MTVs Teen Wolf. Saintgnue has a unique relationship with Duchenne. He played a young man with muscular dystrophy in his break out role on House MD in 2009. Saintgnue talks about his experience learning to mimic the physicality of a young man with Duchenne, as well as the inspiration he draws from the way those young men overcome many obstacles to live happy, fulfilling lives.

Upcoming interviews will feature: Professor Rachelle Crosbie-Watson from the University of California, Los Angeles, who teaches the first university course focused entirely on Duchenne; Dr. Ron Victor, a Cedars-Sinai cardiologist and researcher looking at the benefits of Cialis and Viagra for Duchenne cardiomyopathy; and, Scotty Bob Morgan, a daredevil wingsuit pilot, who has raised awareness worldwide about Duchenne, flying a specially made Coalition Duchenne wingsuit.

About Duchenne muscular dystrophy: Duchenne muscular dystrophy is a progressive muscle wasting disease. It is the most common fatal disease that affects children. Duchenne occurs in 1 in 3,500 male births, across all races, cultures and countries. Duchenne is caused by a defect in the gene that codes for the protein dystrophin. This is a vital protein that helps connect the muscle fiber to the cell membranes. Without dystrophin, the muscle cells become unstable, are weakened and lose their functionality. Life expectancy ranges from the mid teenage years to the mid 20s. Their minds are unaffected.

About Coalition Duchenne: Jayasuriya founded Coalition Duchenne in 2010 (http://www.coalitionduchenne.org) to raise global awareness for Duchenne muscular dystrophy, to fund research and to find a cure for Duchenne. Coalition Duchenne is a 501c3 non-profit corporation.

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Coalition Duchenne Launches Youtube Interview Series 'Making a Difference in Duchenne'

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Stem cell transplant (also known as bone marrow transplant or BMT) is an established treatment for many cancers and blood diseases once considered incurable. For some types of blood diseases, transplantation is the standard of care. For others, it is only considered if other treatments have not been successful. Ongoing advances in stem cell transplant are expanding its availability and improving outcomes for patients, young and old.

Here at the University of Chicago Medicine, the brightest minds in medicine are ready to meet the needs of all patients considering a stem cell transplant. We offer the latest promising approaches in blood and bone marrow stem cell transplant. Our team is known -- and recognized -- for our experience and expertise in:

We provide outstanding and compassionate care in a patient-centered environment. The Stem Cell Transplant Unit, located on the top floor of the Center for Care and Discovery, offers the newest technology as well as many thoughtful patient and family amenities. The unit integrates both inpatient and outpatient stem cell transplant care services in one convenient location.

As part of the internationally recognized University of Chicago Comprehensive Cancer Center (UCCCC), we participate in national clinical trials testing new and emerging therapies. A primary site for early-phase clinical trials, we offer our patients access to more new treatment protocols than any other hospital in the region.

As a leading center for advanced care, the University of Chicago Medicine attracts patients from throughout the region, the country and around the world. We provide customized services for patients who travel from other countries. For more information, contact the Center for International Patients.

In the late 1940s, University of Chicago researcher Dr. Leon Jacobson discovered that he could save a mouse, whose bone marrow and spleen had been destroyed with radiation, by transplanting healthy spleen tissue from another mouse. The donated tissue repopulated the marrow and restored production of the blood cells. This groundbreaking work influenced many scientists investigating bone marrow transplant for humans, including the winner of the 1990 Nobel Prize in Physiology or Medicine.

For information about stem cell transplant for children and teens, visit the Pediatric Stem Cell Transplant page on the University of Chicago Comer Childrens Hospital website.

UCH_008151 (19)

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Blood and Bone Marrow Stem Cell Transplantation - The ...

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Lung diseases like emphysema and pulmonary fibrosis are common among people with malfunctioning telomeres, the "caps" or ends of chromosomes. Now, researchers from Johns Hopkins say they have discovered what goes wrong and why.

Mary Armanios, M.D., an associate professor of oncology at the Johns Hopkins University School of Medicine., and her colleagues report that some stem cells vital to lung cell oxygenation undergo premature aging -- and stop dividing and proliferating -- when their telomeres are defective. The stem cells are those in the alveoli, the tiny air exchange sacs where blood takes up oxygen.

In studies of these isolated stem cells and in mice, Armanios' team discovered that dormant or senescent stem cells send out signals that recruit immune molecules to the lungs and cause the severe inflammation that is also a hallmark of emphysema and related lung diseases.

Until now, Armanios says, researchers and clinicians have thought that "inflammation alone is what drives these lung diseases and have based therapy on anti-inflammatory drugs for the last 30 years."

But the new discoveries, reported March 30 in Proceedings of the National Academy of Sciences, suggest instead that "if it's premature aging of the stem cells driving this, nothing will really get better if you don't fix that problem," Armanios says.

Acknowledging that there are no current ways to treat or replace damaged lung stem cells, Armanios says that knowing the source of the problem can redirect research efforts. "It's a new challenge that begins with the questions of whether we take on the effort to fix this defect in the cells, or try to replace the cells," she adds.

Armanios and her team say their study also found that this telomere-driven defect leaves mice extremely vulnerable to anticancer drugs like bleomycin or busulfan that are toxic to the lungs. The drugs and infectious agents like viruses kill off the cells that line the lung's air sacs. In cases of telomere dysfunction, Armanios explains, the lung stem cells can't divide and replenish these destroyed cells.

When the researchers gave these drugs to 11 mice with the lung stem cell defect, all became severely ill and died within a month.

This finding could shed light on why "sometimes people with short telomeres may have no signs of pulmonary disease whatsoever, but when they're exposed to an acute infection or to certain drugs, they develop respiratory failure," says Armanios. "We don't think anyone has ever before linked this phenomenon to stem cell failure or senescence."

In their study, the researchers genetically engineered mice to have a telomere defect that impaired the telomeres in just the lung stem cells in the alveolar epithelium, the layer of cells that lines the air sacs. "In bone marrow or other compartments, when stem cells have short telomeres, or when they age, they just die out," Armanios says. "But we found that instead, these alveolar cells just linger in the senescent stage."

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Premature aging of stem cell telomeres, not inflammation, linked to emphysema

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Costa Mesa and Sherman Oaks, California (PRWEB) March 31, 2015

The Irvine Stem Cell Treatment Center announces a series of free public seminars on the use of adult stem cells for various degenerative and inflammatory conditions. They will be provided by Dr. Thomas A. Gionis, Surgeon-in-Chief.

The seminars will be held on Wednesday, April 8, 2015, at 11:00 am, 1:00 pm and 3:00 pm at Ayres Hotel & Suites Costa Mesa/Newport Beach, 325 Bristol Street, Costa Mesa, CA 92626; and Wednesday, April 22, 2015, at 11:00 am, 1:00 pm and 3:00 pm at Hampton Inn, 5638 Sepulveda Blvd., Sherman Oaks, CA 91411. Please RSVP at (949) 679-3889.

The Irvine Stem Cell Treatment Center (Irvine and Westlake), along with sister affiliates, the Miami Stem Cell Treatment Center (Miami; Boca Raton; Orlando; The Villages; Sarasota, Florida) and the Manhattan Regenerative Medicine Medical Group (Manhattan, New York), abide by approved investigational protocols using adult adipose derived stem cells (ADSCs) which can be deployed to improve patients quality of life for a number of chronic, degenerative and inflammatory conditions and diseases. ADSCs are taken from the patients own adipose (fat) tissue (found within a cellular mixture called stromal vascular fraction (SVF)). ADSCs are exceptionally abundant in adipose tissue. The adipose tissue is obtained from the patient during a 15 minute mini-liposuction performed under local anesthesia in the doctors office. SVF is a protein-rich solution containing mononuclear cell lines (predominantly adult autologous mesenchymal stem cells), macrophage cells, endothelial cells, red blood cells, and important Growth Factors that facilitate the stem cell process and promote their activity.

ADSCs are the bodys natural healing cells - they are recruited by chemical signals emitted by damaged tissues to repair and regenerate the bodys injured cells. The Irvine Stem Cell Treatment Center only uses Adult Autologous Stem Cells from a persons own fat No embryonic stem cells are used; and No bone marrow stem cells are used. Current areas of study include: Emphysema, COPD, Asthma, Heart Failure, Heart Attack, Parkinsons Disease, Stroke, Traumatic Brain Injury, Lou Gehrigs Disease, Multiple Sclerosis, Lupus, Rheumatoid Arthritis, Crohns Disease, Muscular Dystrophy, Inflammatory Myopathies, and Degenerative Orthopedic Joint Conditions (Knee, Shoulder, Hip, Spine). For more information, or if someone thinks they may be a candidate for one of the adult stem cell protocols offered by the Irvine Stem Cell Treatment Center, they may contact Dr. Gionis directly at (949) 679-3889, or see a complete list of the Centers study areas at: http://www.IrvineStemCellsUSA.com.

Also, you can listen and call into our new radio show, The Stem Cell Show, hosted by Dr. Gionis on TalkRadio 790 AM KABC, Sundays @ 4pm PST, or worldwide on KABC.com ("Listen Live" at 4pm PST) or the KABC app available on the App Store or Google Play.

About the Irvine Stem Cell Treatment Center: The Irvine Stem Cell Treatment Center, along with sister affiliates, the Miami Stem Cell Treatment Center and the Manhattan Regenerative Medicine Medical Group, is an affiliate of the California Stem Cell Treatment Center / Cell Surgical Network (CSN); we are located in Irvine and Westlake, California. We provide care for people suffering from diseases that may be alleviated by access to adult stem cell based regenerative treatment. We utilize a fat transfer surgical technology to isolate and implant the patients own stem cells from a small quantity of fat harvested by a mini-liposuction on the same day. The investigational protocols utilized by the Irvine Stem Cell Treatment Center have been reviewed and approved by an IRB (Institutional Review Board) which is registered with the U.S. Department of Health, Office of Human Research Protection (OHRP); and our studies are registered with Clinicaltrials.gov, a service of the U.S. National Institutes of Health (NIH). For more information, visit our websites: http://www.IrvineStemCellsUSA.com, http://www.MiamiStemCellsUSA.com, or http://www.NYStemCellsUSA.com; http://www.TheStemCellShow.com.

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The Irvine Stem Cell Treatment Center Announces Adult Stem Cell Public Seminars in Costa Mesa and Sherman Oaks ...

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(SACRAMENTO, Calif.) - The state stem cell agency, California Institute for Regenerative Medicine (CIRM),awarded a pair of grants totaling more than $7 million to UC Davis School of Medicine researchers who are working to develop stem cell therapies for spina bifida and chronic diabetic wounds. The funding is part of what the agency considers "the most promising" research leading up to human clinical trials using stem cells to treat disease and injury. Diana Farmer, professor and chair of surgery at UC Davis Medical Center, is developing a placental stem cell therapy for spina bifida, the common and devastating birth defect that causes lifelong paralysis as well as bladder and bowel incontinence. She and her team are working on a unique treatment that can be applied in utero - before a baby is born -- in order to reverse spinal cord damage. Roslyn Rivkah Isseroff, a UC Davis professor of dermatology, and Jan Nolta, professor of internal medicine and director of the university's Stem Cell Program, are developing a wound dressing containing stem cells that could be applied to chronic wounds and be a catalyst for rapid healing. This is Isseroff's second CIRM grant, and it will help move her research closer to having a product approved by the U.S. Food and Drug Administration that specifically targets diabetic foot ulcers, a condition affecting more than 6 million people in the country. The CIRM board, which met in Berkeley today, has high hopes for these types of research that the agency funded in this latest round of stem cell grants. "This investment will let us further test the early promise shown by these projects," said Jonathan Thomas, chair of CIRM's governing board. "Preclinical work is vital in examining the feasibility, potential effectiveness and safety of a therapy before we try it on people. These projects all showed compelling evidence that they could be tremendously beneficial to patients. We want to help them build on that earlier research and move the projects to the next level." The CIRM grants are designed to enable the UC Davis research teams to transition from preclinical research to preclinical development over the next 30 months to be able to meet the FDA's rigorous safety and efficacy standards for Investigative New Drugs. As the former surgeon-in-chief at UCSF Benioff Children's Hospital, Farmer helped pioneer fetal surgery techniques for treating spina bifida before birth. The condition, also known as myelomeningocele, is one of the most common and devastating birth defects worldwide, causing lifelong paralysis as well as bowel and bladder incontinence in newborns. Farmer has been investigating different stem cell types and the best way to deliver stem cell-based treatments in the womb for the past six years. She and her research colleagues recently discovered a placental therapy using stem cells that cures spina bifida in animal models. That discovery requires additional testing and FDA approval before the therapy can be used in humans. With the CIRM funding, Farmer and her team plan to optimize their stem cell product, validate its effectiveness, determine the optimal dose and confirm its preliminary safety in preparation for human clinical trials. Isseroff, who also serves as chief of dermatology and director of wound healing services for the VA Northern California Health Care System, has long been frustrated by the challenges of treating the chronic, non-healing wounds of diabetics. In 2010, she and Nolta received a CIRM grant to begin developing a bioengineered product for treating chronic diabetic wounds. Foot ulcers, in particular, affect about 25 percent of all diabetic patients and are responsible for most lower-limb amputations. Isseroff and her research team created a treatment using stem cells derived from bone marrow (mesenchymal stem cells) along with a FDA-approved scaffold to help regenerate dermal tissue and restart the healing process. Their studies found the technique to be highly effective for healing wounds in animal models. With this latest CIRM grant, Isseroff's team will refine their therapeutic technique by determining the safest dosage for regenerating tissue and testing their product in skin-wound models that closely resemble those in diabetic humans. Nolta also plans to create a Master Cell Bank of pure and effective human mesenchymal stem cells, and establish standard operating procedures for use in diabetic wound repair. The results of their efforts will enable UC Davis to move closer to FDA approval for human clinical trials in the next two and a half years. "These amazing research efforts are giant steps forward in turning stem cells into cures," said Nolta, who also directs the UC Davis Institute for Regenerative Cures in Sacramento. "This preclinical research is the most crucial, and often the toughest, stage before we move scientific discoveries from the laboratory bench to the patient's bedside. We are now poised as never before to make a big difference in the lives of people with spina bifida and non-healing diabetic wounds." For more information, visit UC Davis School of Medicine at http://medschool.ucdavis.edu.

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Stem Cell Grants for Spina Bifida and Diabetic Wound Treatments

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Two approaches to fat grafting -- injection of fat cells versus fat-derived stem cells -- have similar effects in reversing the cellular-level signs of aging skin, reports a study in the April issue of Plastic and Reconstructive Surgery, the official medical journal of the American Society of Plastic Surgeons (ASPS).

Since the facial rejuvenation results are the same, the simpler approach using fat cells plus the "stromal vascular fraction" has advantages over the more time-consuming stem cell fat technique. Dr. Gino Rigotti of Clinica San Francesco, Verona, Italy, directed a research team consisting of Luiz Charles-de-S and Natale Ferreira Gontijo-de-Amorim from Clinica Performa, Rio de Janeiro; and Andrea Sbarbati, Donatella Benati, and Paolo Bernardi from the Anatomy and Histology Institute, University of Verona.

Fat Grafts vs Stem Cells for Facial Rejuvenation

The experimental study compared the two approaches to fat grafting for regeneration of the facial skin. In these procedures, a small amount of the patient's own fat is obtained by liposuction from another part of the body, such as the abdomen. After processing, the fat is grafted (transplanted) to the treated area, such as the face.

The study included six middle-aged patients who were candidates for facelift surgery. All underwent fat grafting to a small area in front of the ear.

One group of patients received fat-derived stem cells. Isolated and grown from the patients' fat, these specialized cells have the potential to develop into several different types of tissue. The other group underwent injection of fat cells along with the stromal vascular fraction (SVF) -- a rich mix of cell types, including stem cells.

Before and three months after fat grafting, samples of skin from the treated area were obtained for in-depth examination, including electron microscopy for ultrastructural-level detail.

After injection of fat cells plus SVF, the skin samples showed reduced degeneration of the skin's elastic fiber network, or "elastosis" -- a key characteristic of aging skin. These findings were confirmed by ultrastructural examination, which demonstrated the reabsorption of the elastosis and the development of relatively "young" elastic fibers.

In patients undergoing stem cell injection, the skin changes were essentially identical. "This result seems to suggest that the effect of a fat graft is, at least in part, due to its stem cell component," Dr. Rigotti and coauthors write.

The researchers also found "suggestive" evidence that the rejuvenating effects of fat grafting are related to new formation of microscopic blood vessels. Further studies are needed to confirm this hypothesis, however. Dr. Rigotti comments, "In any case, this is the first study presenting clinical evidence showing skin rejuvenation after fat grafting and highlighting the anatomical and structural changes that are the basis of this rejuvenation."

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Two different fat graft techniques have similar effects on facial skin

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$1 M gift from Mr. J. Sebastian van Berkom launches translational research into neurological disease

This news release is available in French.

A patient's very own skin cells may hold the key to new treatments and even cures for devastating neurological diseases. A generous $1 million donation from Mr. J. Sebastian van Berkom, and critical partnerships with Brain Canada, Laval University, Marigold Foundation and the FRQS-Rseau Parkinson Quebec are driving an innovative, iPSC (induced pluripotent stem cell) research platform that will transform research into Parkinson's and other neurological diseases.

Millions of Canadians are affected by diseases of the brain such as ALS, Parkinson's and brain tumours, for which there are limited treatments and no cures. By 2020, neurological conditions will become the leading cause of death and disability. "Everyone's lives are touched in some way by neurological disease, says Mr. van Berkom, President of Van Berkom and Associates Inc." In creating The van Berkom Parkinson's Disease Open-Access Fund, I hope to change lives and support new research that will lead to new treatments and one day cures. The iPSC platform is a new paradigm for neuroscience research and as one of the world's great neuroscience centres, The Neuro is the place to drive it forward."

"This is the ultimate bench to bedside paradigm, from patient to the bench, back to the patient," says Dr. Guy Rouleau, Director of The Neuro. "With a unique interface between fundamental and clinical research, The Neuro is uniquely positioned to be a central hub in the iPSC platform. Partnering with Mr. Van Berkom, a generous and visionary philanthropist, propels The Neuro toward the goal of significantly deepening insight into disease mechanisms with unprecedented efficiency."

Patients' skin cells will be reprogrammed into induced pluripotent stem cells (iPSCs) at Laval University, under the leadership of Dr Jack Puymirat, and then differentiated at The Neuro into disease relevant cells for research. For example, in the case of Parkinson's this could be dopamine neurons. The cells can also be genome-edited, a state-of-the-art technique that can introduce or correct disease associated mutations - creating the most accurate disease models. These iPSCs will be made widely and openly available to researchers across Quebec for neuroscience research. This open-access approach exponentially increases the likelihood of breakthroughs in neurological disease.

"The unique and exciting aspect of this platform is that we are creating the most specific cells for studying disease using the patient's own tissue, which has distinct advantages over using generic cells or animal models," says Dr. Edward Fon, neurologist and co-Director of the Quebec iPSC platform. "Disease models using human samples are increasingly shown to be far more efficacious in trials, as they much more accurately mimic the disease condition. In the iPSC platform, not only can specific mutations be introduced but, cells are from patients' whose specific clinical history and genetic profile are known, a first step on the road toward neurological personalized medicine. The Neuro has access to a large and well-characterized patient population, who can help create a rich clinically-and genetically-derived registry and biobank. The initial targets in the platform will be ALS and Parkinson's disease (PD), using dopamine neurons for PD and both motor neurons and astrocytes for ALS."

The Quebec iPSC core facility is a provincial core headed by Drs. Fon and Puymirat. Reprogrammed cells at Laval University will be created from different sources such as skin biopsies, blood or urine. The Neuro's component of the platform will consist of two core facilities. The iPSC neuronal differentiation core - which differentiate iPSCs into functional neurons, headed by Dr. Eric Shoubridge, and the iPSC genome-editing core providing unprecedented ability to study the influence of disease mutations, headed by Dr. Peter McPherson.

###

The Montreal Neurological Institute and Hospital

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Using patients' own cells to accelerate research into neurological disease

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Durham, NC (PRWEB) March 31, 2015

Stem cells may provide Crohns disease sufferers relief from a common, potentially dangerous side effect fistulas according to the results of a phase 2 clinical trial published in the latest issue of STEM CELLS Translational Medicine (SCTM). After receiving an injection of their own adipose-derived stem cells (ASC), which are collected from fat tissue, the fistulas in 75 percent of the trial participants were completely healed within eight weeks of their last treatment and remained so two years later.

Crohn's disease is a painful, chronic autoimmune disorder in which the body's immune system attacks the gastrointestinal tract. Inflammation in Crohns patients can sometimes extend completely through the intestinal wall and create a fistula an abnormal connection between the intestine and another organ or skin. Left untreated, a fistula might become infected and form an abscess, which in some cases can be life threatening.

Chang Sik Yu, M.D., Ph.D., of Asan Medical Center in Seoul, Korea, a senior author of the SCTM paper, describes the results of a clinical trial conducted in collaboration with four other hospitals in South Korea, stated, Crohns fistula is one of the most distressing diseases as it decreases patients quality of life and frequently recurs. It has been reported to occur in up to 38 percent of Crohns patients and over the course of the disease, 10 to 18 percent of them must undergo a proctectomy, which is a surgical procedure to remove the rectum.

Overall, the treatments currently available for Crohns fistula remain unsatisfactory because they fail to achieve complete closure, lower recurrence and limit adverse effects, Dr. Yu said. Given the challenges and unmet medical needs in Crohns fistula, attention has turned to stem cell therapy as a possible treatment.

Several studies, including those undertaken by Dr. Yus team, suggest that mesenchymal stem cells (MSCs) do indeed improve Crohns disease and Crohns fistula. Their phase II trial involved 43 patients for a term of one year, over the period from January 2010 to August 2012. The results showed that 82 percent experienced complete closure of fistula eight weeks after the final ASC injection.

It strongly demonstrated MSCs derived from ASCs are a safe and useful therapeutic tool for the treatment of Crohns fistula, Dr. Yu said.

The latest study was intended to evaluate the long-term outcome by following 41 of the original 43 patients for yet another year. Dr. Yu reported, Our long-term follow-up found that one or two doses of autologous ASC therapy achieved complete closure of the fistulas in 75 percent of the patients at 24 months, and sustainable safety and efficacy of initial response in 83 percent. No adverse events related to ASC administration were observed. Furthermore, complete closure after initial treatment was well sustained.

These results strongly suggest that autologous ASCs may be a novel treatment option for Crohns fistulae, he said.

Stem cells derived from fat tissue are known to regulate the immune response, which may explain these successful long-term results treating Crohns fistulae with a high risk of recurrence, said Anthony Atala, M.D., Editor-in-Chief of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine.

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Trial Shows Stem Cells Provide Long-Term Relief from Dangerous Crohns Side Effect

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Testimonial John Edwards
John Edwards of Roatan provides a testimonial for his orthopedic stem cell treatments recently at GARM.

By: Global Alliance for Regenerative Medicine, Roatan, Honduras

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Testimonial John Edwards - Video

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Treatment of hip tendinitis and bursitis
Siddharth Tambar MD, the physician at Chicago Arthritis, discusses regenerative medicine treatments for hip tendinitis and bursitis including the Regenexx platelet rich plasma treatment.

By: Chicago Arthritis

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Treatment of hip tendinitis and bursitis - Video

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Jason Wertheim Bioengineered Kidney and Future of Regenerative Medicine
Skype conversation with Jason Wertheim on "Bioengineered Kidney and Future of Regenerative Medicine" on March 24th, 2015 in the Technology and Future of Medicine course LABMP 590 ...

By: Kim Solez

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Jason Wertheim Bioengineered Kidney and Future of Regenerative Medicine - Video

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What are bone marrow and hematopoietic stem cells?

Bone marrow is the soft, sponge-like material found inside bones. It contains immature cells known as hematopoietic or blood-forming stem cells. (Hematopoietic stem cells are different from embryonic stem cells. Embryonic stem cells can develop into every type of cell in the body.) Hematopoietic stem cells divide to form more blood-forming stem cells, or they mature into one of three types of blood cells: white blood cells, which fight infection; red blood cells, which carry oxygen; and platelets, which help the blood to clot. Most hematopoietic stem cells are found in the bone marrow, but some cells, called peripheral blood stem cells (PBSCs), are found in the bloodstream. Blood in the umbilical cord also contains hematopoietic stem cells. Cells from any of these sources can be used in transplants.

What are bone marrow transplantation and peripheral blood stem cell transplantation?

Bone marrow transplantation (BMT) and peripheral blood stem cell transplantation (PBSCT) are procedures that restore stem cells that have been destroyed by high doses of chemotherapy and/or radiation therapy. There are three types of transplants:

Why are BMT and PBSCT used in cancer treatment?

One reason BMT and PBSCT are used in cancer treatment is to make it possible for patients to receive very high doses of chemotherapy and/or radiation therapy. To understand more about why BMT and PBSCT are used, it is helpful to understand how chemotherapy and radiation therapy work.

Chemotherapy and radiation therapy generally affect cells that divide rapidly. They are used to treat cancer because cancer cells divide more often than most healthy cells. However, because bone marrow cells also divide frequently, high-dose treatments can severely damage or destroy the patients bone marrow. Without healthy bone marrow, the patient is no longer able to make the blood cells needed to carry oxygen, fight infection, and prevent bleeding. BMT and PBSCT replace stem cells destroyed by treatment. The healthy, transplanted stem cells can restore the bone marrows ability to produce the blood cells the patient needs.

In some types of leukemia, the graft-versus-tumor (GVT) effect that occurs after allogeneic BMT and PBSCT is crucial to the effectiveness of the treatment. GVT occurs when white blood cells from the donor (the graft) identify the cancer cells that remain in the patients body after the chemotherapy and/or radiation therapy (the tumor) as foreign and attack them. (A potential complication of allogeneic transplants called graft-versus-host disease is discussed in Questions 5 and 14.)

What types of cancer are treated with BMT and PBSCT?

BMT and PBSCT are most commonly used in the treatment of leukemia and lymphoma. They are most effective when the leukemia or lymphoma is in remission (the signs and symptoms of cancer have disappeared). BMT and PBSCT are also used to treat other cancers such as neuroblastoma (cancer that arises in immature nerve cells and affects mostly infants and children) and multiple myeloma. Researchers are evaluating BMT and PBSCT in clinical trials (research studies) for the treatment of various types of cancer.

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Blood-Forming Stem Cell Transplants - National Cancer ...

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IMAGE:Mutations (*) in several Krebs cycle genes (IDH1/2, SDH, FH and now MDH2) lead to the accumulation of metabolites (2-HG, SUC and FUM), which cause important changes in gene expression... view more

Credit: CNIO

Researchers in the Hereditary Endocrine Cancer Group of the Spanish National Cancer Research Centre (CNIO) -- led by Alberto Cascn and Mercedes Robledo -- have described the presence of mutations in the MDH2 gene, in a family with very rare neuroendocrine tumours associated with a high hereditary component: pheochromocytomas and paragangliomas that affect the suprarenal and parathyroid glands (groups of chromaffin cells in the central nervous system), respectively. This research has been published in the Journal of the National Cancer Institute.

Pheochromocytomas and paragangliomas are rare diseases, with an incidence of 3 to 8 cases per million inhabitants. In spite of this low incidence, they represent a paradigm in hereditary cancer because they are the tumours with the highest hereditary predisposition: approximately 50% of the patients inherit and/or transmit the susceptibility to developing this cancer.

To date, researchers had identified 11 main genes whose mutations are responsible for pheochromocytomas and paragangliomas. Of these, 6 are involved in cellular metabolism and more specifically, the Krebs cycle, which is the machinery used by cells to burn oxygen and obtain the energy required for cellular work.

CELLULAR METABOLISM AND CANCER

The researchers sequenced the whole exome -- the part of the genome that produces the proteins -- of one of the tumours in a patient with multiple malignant pheochromocytomas and paragangliomas; this tumour showed no mutations in any of the 11 genes associated with genetic susceptibility to developing the disease.

Analysis and filtering of the almost 80,000 variants found in the sample allowed the identification of a mutation in the MDH2 gene, whose association with this type of cancer had not been described before. Moreover, "the presence of the mutation in first-degree relatives, one of whom was subsequently diagnosed with the disease, confirmed the hereditary nature of this genetic alteration," explains Cascn.

The new discovery confirms the relationship between metabolism and the development of this type of tumour. Mutations in the Krebs cycle genes in patients with these tumours cause a metabolic alteration that leads to the accumulation of specific metabolites. These metabolites, known as oncometabolites, give rise to epigenetic changes in the genome that cause global gene expression changes and, as a consequence, the appearance of tumours.

IMPROVED GENETIC DIAGNOSIS

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The CNIO identifies a new gene involved in hereditary neuroendocrine tumors

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Illuminati Science EXPOSED Genetic Engineering, Cloning, DNA Manipulation, Transhumanism 1080p
I am just a middleman trying to spread the word FAIR USE NOTICE: This video may contain copyrighted material. Such material is made available for entertainment purposes only. This constitutes...

By: Timothy anon

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Illuminati Science EXPOSED Genetic Engineering, Cloning, DNA Manipulation, Transhumanism 1080p - Video

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What is Genetic Engineering? - by Wideo.co
This video was created using wideo.co, the best online tool to easily create animated videos. Make your own video now at http://goo.gl/X153KO.

By: Sruthi J

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What is Genetic Engineering? - by Wideo.co - Video

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