Category Archives: news

Stem cell gene therapy developed at UCLA holds promise for eliminating HIV infection

Method modifies immune cells to attack the virus, could be used to treat other diseases

Scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research are one step closer to engineering a tool that could one day arm the body’s immune system to fight HIV — and win. The new technique harnesses the regenerative capacity of stem cells to generate an immune response to the virus.

The findings were published today in the journal Molecular Therapy.

Protein plays unexpected role in embryonic stem cells

A protein long believed to only guard the nucleus also regulates gene expression and stem cell development

LA JOLLA – What if you found out that pieces of your front door were occasionally flying off the door frame to carry out chores around the house? That’s the kind of surprise scientists at the Salk Institute experienced with their recent discovery that nucleoporins—proteins that act as cellular “doorways” to help manage what goes in and out of a cell’s nucleus—are actually much bigger players in expressing genes than previously thought.

The finding, published June 16, 2015 in the journal Genes & Development, shows that nucleoporins play an important role in maintaining embryonic stem cells before they begin to develop into specific tissues. This discovery gives a new understanding to genetic diseases that are caused by mutations in these proteins. One nucleoporin protein in particular has a dramatic—and unanticipated—function in the formation of neurons from stem cells.

Stem Cell Injection May Soon Reverse Vision Loss Caused By Age-Related Macular Degeneration

An injection of stem cells into the eye may soon slow or reverse the effects of early-stage age-related macular degeneration, according to new research from scientists at Cedars-Sinai. Currently, there is no treatment that slows the progression of the disease, which is the leading cause of vision loss in people over 65.

“This is the first study to show preservation of vision after a single injection of adult-derived human cells into a rat model with age-related macular degeneration,” said Shaomei Wang, MD, PhD, lead author of the study published in the journal STEM CELLS and a research scientist in the Eye Program at the Cedars-Sinai Board of Governors Regenerative Medicine Institute.

The stem cell injection resulted in 130 days of preserved vision in laboratory rats, which roughly equates to 16 years in humans.

Cardiac Stem Cell Therapy May Heal Heart Damage Caused by Duchenne Muscular Dystrophy

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.

Remotely controlled magnetic nanoparticles stimulate stem cells to regenerate better, denser bone

Durham, NC – In a recent study published in STEM CELLS Translational Medicine, scientists have found what they believe might be a better way to regrow bone tissue using magnetic nanoparticles coated with targeting proteins that stimulate stem cells to regenerate the bone. They also were able to deliver the cells directly to the injured area, remotely control the nanoparticles to generate mechanical forces and maintain the regeneration process through staged releases of a protein growth factor. These findings might someday have significant impact for anyone suffering from a major bone trauma, disease or defect.

The gold standard for repairing bone that can’t heal itself is a graft taken from the patient. Unfortunately this is a painful, invasive procedure, plus there are times when the area that needs repair is too massive or the patient has a skeletal disorder so has no healthy bone for grafting. That’s why spurring the growth of new bone through injected stem cells is an area of great interest to medical researchers. Much progress has been made to this end, but a major hurdle remains – finding an appropriate means by which to stimulate the differentiation of the stem cells so they become the quality of bone tissue needed in a quantity large enough to treat patients effectively.

TechKnow: Banking cord blood to save lives

This Saturday, July 19th at 7:30 pm ET/4:30 pm PT (Repeats 10:30P ET/7:30P PT), Al Jazeera America’s “TechKnow” shows us one of the most exiting areas of medical research – how umbilical cord blood is being used to treat brain disease and injury.

“Techknow” host and mechanical engineer Dr. Shini Somara takes us inside Duke Children’s Hospital, where a team of doctors is treating young Grace Matthews, an infant with hydrocephalus, or water on the brain, characterized by the tell-tale swelling of the baby’s head. We go behind-the-scenes on the high-tech experimental treatments, as doctors infuse Grace with stem cells from her own umbilical cord, and we meet another young patient who experienced “miraculous” progress from the use of umbilical cord stem cells.

A Changing View of Bone Marrow Cells

Caltech researchers show that the cells are actively involved in sensing infection.

RESEARCHERS SEPARATE BLOOD STEM CELLS FROM OTHER BONE MARROW CELLS AND LOAD THEM ONTO A NEW MICROFLUIDIC CHIP. FLUORESCENT SIGNALS INDICATE THE PRESENCE OF SECRETED PROTEINS WITH ONE "BARCODE" REPRESENTING EACH CELL.

RESEARCHERS SEPARATE BLOOD STEM CELLS FROM OTHER BONE MARROW CELLS AND LOAD THEM ONTO A NEW MICROFLUIDIC CHIP. FLUORESCENT SIGNALS INDICATE THE PRESENCE OF SECRETED PROTEINS WITH ONE “BARCODE” REPRESENTING EACH CELL.

In the battle against infection, immune cells are the body’s offense and defense—some cells go on the attack while others block invading pathogens. It has long been known that a population of blood stem cells that resides in the bone marrow generates all of these immune cells. But most scientists have believed that blood stem cells participate in battles against infection in a delayed way, replenishing immune cells on the front line only after they become depleted.

Now, using a novel microfluidic technique, researchers at Caltech have shown that these stem cells might be more actively involved, sensing danger signals directly and quickly producing new immune cells to join the fight.

“It has been most people’s belief that the bone marrow has the function of making these cells but that the response to infection is something that happens locally, at the infection site,” says David Baltimore, president emeritus and the Robert Andrews Millikan Professor of Biology at Caltech. “We’ve shown that these bone marrow cells themselves are sensitive to infection-related molecules and that they respond very rapidly. So the bone marrow is actually set up to respond to infection.”

Stem cell therapy shows promise in repairing brain damage even hours after stroke occurs

Durham, NC – Stroke is a major health concern and is a leading cause of death in the United States, according to the Center for Disease Control. Despite significant research efforts, developing treatments that ensure complete recovery for stroke patients poses an extreme challenge, especially when more than a few hours have passed between onset of the stroke and administration of treatment.

However, a new study released today in STEM CELLS Translational Medicine indicates that endothelial precursor cells, which are found in the bone marrow, umbilical cord blood, and as very rare cells in peripheral blood, could make a significant difference for these patients’ recovery — even in the later stages of stroke. In animal studies, the treatment minimized the initial brain injury and helped repair the stroke damage.

Stem Cells, Cell and Gene Therapy Biotechs to Discuss Capital Formation Strategies and Validation

NEW YORK – In a progressive cell therapy environment that has decreasing clinical attrition, next generation and next-to-launch biotechs along with regulators, will be talking about current strategies and approaches to advance the development of stem cells, cell and gene therapies.

Over 250 biotechs, biopharma, research institutions and regulators will gather at the Stem Cells USA & Regenerative Medicine Congress on September 30 – October 1, 2013.

Novel liver stem cell model could speed up process for developing new drugs

Durham, NC – The path to bringing a new drug to market is, simply put, a rocky one. Not only is it estimated to take over 12 years at an average price tag running anywhere between US $800 million and US $2 billion, but more often than not the new drug never makes it through the process.

But now a research team reports that it has developed a way to speed up the process. Their work, which involves the creation of a highly stable and sensitive liver stem cell model, is reported in the latest issue of STEM CELLS Translational Medicine.

“Liver toxicity is the second most common cause of human drug failure,” explained David Hay, Ph.D., of the University of Edinburgh’s MRC Centre for Regenerative Medicine, who led the team made up of university colleagues and scientists from Bristol-Myers Squibb, Princeton, N.J. “But one major bottleneck in safety testing new drugs has been finding a routine supply of good quality primary human hepatocytes from the desired genetic background.”

Scientists have long believed that finding an efficient way to force pluripotent stem cells (PSCs) to develop into hepatocytes — liver cells — could be the way around the problem. “But faithfully recapitulating human physiology in a dish from a renewable source remains a holy grail for medicine and the pharmaceutical industry,” Dr. Hay noted.

“Many procedures have been described that, to a limited extent, exhibit human-tissue-specific function in vitro but incomplete cellular differentiation and/or the loss of cell phenotype after they differentiate. Using our knowledge in pharmacology, stem cell biology and materials chemistry, we developed a highly stable and sensitive model.”

Their method involved expanding PSCs and driving their differentiation to hepatocytes, then replating them onto a synthetic surface. The results yielded active cell populations that displayed stable function for over two weeks in vitro.

“The scalable nature of our model combined with the interchangeable genetic element demonstrates clear advantages over the erratic supply of highly variable human hepatocytes from deceased specimens,” Dr. Hay added. “We believe our approach is important and will likely contribute to improvements in drug safety testing.”

“This model was compared to human liver cells from deceased donors and found to be equivalent, suggesting that stem cell-derived hepatocyles have potential to improve the preclinical assessment of human liver toxicity,” said Anthony Atala, M.D., Editor of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine.”

The full article, “Developing high fidelity hepatotoxicity models from pluripotent stem cells,” can be accessed at http://www.stemcellstm.com