UCLA Stem Cell Researchers Announce Gene Therapy Cure for 18 Bubble Baby Patients Researchers at UCLA announced today that they had cured 18 children who were born with the so-called Bubble Baby disease, a genetic disorder that leaves the young sufferers without a working... By: UCLA Eli Edythe Broad Center of Regenerative Medicine and Stem Cell Research … Continue reading →

Copy of HOPE, HYPE REALITY: ZINC FINGER GENE THERAPY TO CONTROL OR CURE HIV A video to answer your questions about a gene therapy approach to an HIV cure. On Nov 5, 2014, the defeatHIV Community Advisory Board hosted a talk with Dr. ... By: defeatHIV … Continue reading →

Gene therapy restores rare genetic blindness By: cea aery … Continue reading →

Gene Therapy - Treating the Disease Instead of the Symptoms | Tomorrow Today Children found to be particularly susceptible to illness could be suffering from a genetic - and potentially fatal - immune disorder. This year's prestigious Robert Koch Prize has been awarded... By: DW (English) … Continue reading →

PHILADELPHIA - In bright daylight, 10-year-old Mark DeVoe has no trouble seeing his friends, but inside, or even in the shade, Mark's eyes sometimes don't work. "I have trouble seeing like, trees, when the road ends, and when there's like a drop there," Mark said. At age six, Mark's doctors diagnosed him with the genetic condition choroideremia, which causes people to progressively lose vision until they are completely blind. "I don't know what it's like to live in darkness, but I've seen it," said Susan DeVoe, Mark's mother. Susan is a carrier of the blindness gene. Mark's grandfather has the condition. "Watching my father go blind was devastating. I was a little girl. You know, you count on daddy to do things, and daddy couldn't do them," she recalled. Dr. Jean Bennett is one of two U.S. researchers preparing to test a gene therapy for choroideremia in humans. "I think gene therapy holds a huge promise for developing treatments for blinding diseases," said Bennett, ophthalmologist and molecular geneticist at the University of Pennsylvania. Researchers will use a virus, carrying a normal choroideremia gene and inject the virus just under the retina. The gene should begin to work in a few … Continue reading →

Medgenics, a Wayne-based firm developing gene therapies in cooperation with Childrens Hospital of Philadelphia to treat kidney anemia and other blood problems using a patient's own tissue, says it has raised at least $21 million in a public stock offering, selling 5.125 million shares at $4.10/share, with additional shares available to Piper Jaffray & Co., JMP Securities and other brokers that led the sale. The stock, listed on the New York Stock Exchange (MDGN) since 2011, has been thinly traded. The sale price was a fraction of the $15 Medgenics reached at its 2012 peak; discounts aren't unusual for biotech shares in this market, noted Brian Piper, vice president for finance, told me. The company employs about 7 in the U.S., 33 in an Israel lab, and plans to expand its Philadelphia-area clinical development group. Medgenics's largest shareholder, Isaac Blech, is based in New York. Chairman Sol Barer is also the founder and past CEO of North Jersey-based cancer-fighting therapy maker Celgene (CELG). The proceeds, which Medgenics expects to collect after Dec. 1, will be used to develop the company's TARGT gene therapy platform, including TARGTPRO for renal anemia and hematological conditions, TARGTGLP-2 for short-bowel syndrome, the firm's research and … Continue reading →