News Search: Gene Therapy

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AMD & Stargardt's Disease- New Clinical Studies

Gene therapy, stem cell therapy and vitamin A are being clinically tested as potential cures for improved vision in people suffering from age related macular degeneration and Stargardt’s disease.

It’s a fundamental breakthrough,” said Paul Sieving, director of the National Eye Institute.

Nearly 200 genes involved in vision loss have been identified and biochemical interactions have been explained.

U.S. and Canada Embark on Project to Develop a Gene Therapy for Retinal Blindness

The goal of this project is to begin testing a new gene therapy in patients who are losing their vision due to retinal disease, by the end of five years,” said Dr. Tsilflidis, Senior Scientist at the Ottawa Hospital Research Institute and Associate Professor in the Faculty of Medicine at the University of Ottawa.  By blocking a process (apoptosis) that causes retinal cell death, thus blindness, the XIAP gene therapy protects the retinal cells.  The gene is delivered to the eye using a virus.

Stem Cells and AMD

Research at the Casey Eye Institute at Oregon Health & Science University in Portland showed that putting stem cells near the retinas of rats, that had a form of macular degeneration, helped delay the disease.  Ray Lund, researcher, said that he suspects that when immature nerve cells are placed near the retina, they produce growth factors that protect the cells from damage.  This may protect some people from developing wet AMD as well.

Stem Cells for Reversing Blindness

An Italian study using the person's own limbal corneal stem cells to grow new corneal tissue has resulted in restoring sight to a recipient who had been blind for 50 years.  The ability to grow new corneas opens the possibility to grow new retinal tissue, reversing blindness, providing nerve cells have not been damaged.

The treatment restored sight to more than three-quarters of the 112 patients treated, said lead researcher,  Dr. Graziella Pelligrini of the University of Modena’s Center for Regenerative Medicine. Patients were followed for 3-10 years.

Gene Therapy Cures Color Blindness- AMD Next?

Gene therapy has cured color blindness in monkeys, say researchers from the University of Washington and the University of Florida College of Medicine. They have shown that they can safely cure a cone disease in a primate. Five weeks after the genes were transferred by a harmless adeno-associated virus, the monkeys began to acquire color vision. Potentially, this could help AMD by rescuing targeted cone cells.

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New Tool in Gene Therapy Arsenal

Researchers at Tufts University School of Medicine and the Sackler School of Graduate Biomedical Sciences at Tufts have discovered a new way to transfer DNA, thus safely protecting cells from retinal degeneration. A peptide called PEG-POD is the vehicle for therapeutic gene delivery.