Posted on August 24, 2012

The final installment of a $70,000 grant payable over 2 years for support of research involving gene-based therapeutic interventions for inherited retinal diseases that affect children and young adults. Researchers at Columbia University have identified the gene which causes Stargardt Disease, a juvenile form of macular degeneration, bringing hope that a treatment will eventually be available. This grant is focused on the development of lentiviral vectors to deliver therapeutic genes to the retina. These genes show great promise in gene therapy because they can permanently integrate new genes into the DNA of the cell. The gene therapies are currently being studied in pre-clinical settings with the goal of moving them into human clinical trials as soon as possible.