Latest Gene therapy Stories

A new gene therapy method developed by University of Florida researchers has the potential to treat a common form of blindness that strikes both youngsters and adults. The technique works by replacing a malfunctioning gene in the eye with a normal working copy that supplies a protein necessary for light-sensitive cells in the eye to function. The findings are published today (Monday, Jan. 23) in the Proceedings of the National Academy of Sciences online. Several complex and costly steps...

Members of a University of Pennsylvania research team have shown that they can prevent, or even reverse, a blinding retinal disease, X-linked Retinitis Pigmentosa, or XLRP, in dogs. The disease in humans and dogs is caused by defects in the RPGR gene and results in early, severe and progressive vision loss. It is one of the most common inherited forms of retinal degeneration in man. "Every single abnormal feature that defines the disease in the dogs was corrected following treatment,"...

BOTHELL, Wash., Jan. 23, 2012 /PRNewswire/ -- BioLife Solutions, Inc. (OTCBB: BLFS), a leading developer, manufacturer, and marketer of proprietary hypothermic storage and cryopreservation freeze media for cells, tissues, and organs, today announced that three additional key opinion leaders in the biobanking, drug discovery, and regenerative medicine markets have joined the Company's Scientific Advisory Board (SAB). The new members are: Edward LeCluyse, Ph.D., Senior Research...

1 of the top 10 most common forms of cancer in men and women in the US Bladder cancer, most frequently caused by smoking and exposure to carcinogens in the workplace, is one of the top 10 most common forms of cancer in men and women in the U.S. More than 70 percent of bladder cancers are diagnosed in stage T1 or less and have not invaded the muscle layer. At these early stages, standard treatment is surgery (transurethral resection) and the burning away of tumors with high energy...

In the two and a half years since Adam Bogdanove, professor at Iowa State University in the Department of Plant Pathology and Microbiology, along with Matthew Moscou, a former graduate student in that department, discovered how a class of proteins from plant pathogenic bacteria find and bind specific sequences in plant genomes, researchers worldwide have moved fast to use this discovery. Last year it was first shown that the proteins can be fused to DNA modifying enzymes to manipulate...

Understanding the structure of the TAL effector may be key for targeted gene correction Researchers at Fred Hutchinson Cancer Research Center have solved the three-dimensional structure of a newly discovered type of gene-targeting protein that has shown to be useful as a DNA-targeting molecule for gene correction, gene therapy and gene modification. The findings are published online in Science Express on Jan. 5. Using a unique form of computational and X-ray crystallographic analyses, a...

NEW YORK, Jan. 4, 2012 /PRNewswire/ -- NeoStem, Inc. (NYSE Amex: NBS) ("NeoStem" or the "Company"), an international biopharmaceutical company with a focus on cell-based therapeutic development, today announced that the Company and Progenitor Cell Therapy ("PCT"), will be presenting at four upcoming January conferences: Biotech Showcase 2012, BOCEMb 2012 - Noble Financial Capital Markets Equity Conference, ECI's Inaugural Scale-Up and Manufacturing of Cell-Based Therapies, and...

AMSTERDAM, January 4, 2012 /PRNewswire/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its gene therapy program for the treatment of hemophilia B. Orphan designation in the U.S. could provide up to seven years market exclusivity on regulatory approval. Orphan designation for AMT's hemophilia program in the European Union was...

Diabetes is a major risk factor for cardiovascular disease and can reduce blood supply to the heart tissue and damage cardiac cells, resulting in heart failure. New research has investigated if nerve growth factor (NGF) gene therapy can prevent diabetic heart failure and small vascular disease in mice. The study by Professor Costanza Emanueli, British Heart Foundation Senior Research Fellow and colleagues of the Bristol Heart Institute in the Regenerative Medicine Section of the School of...

Clinical gene therapy may be one step closer, thanks to a new twist on an old class of molecules. A group of University of Illinois researchers, led by professors Jianjun Cheng and Fei Wang, have demonstrated that short spiral-shaped proteins can efficiently deliver DNA segments to cells. The team published its work in the journal Angewandte Chemie. "The main idea is these are new materials that could potentially be used for clinical gene therapy," said Cheng, a professor of materials...