Thanks to the results of a recent MVRF funded study conducted by a team of investigators led by Dr. Robert Molday at the University of British Columbia and Dr. William Hauswirth at the University of Florida, the field of retinal disease has become clearer. They have successfully restored visual function by delivering the normal RD3 gene to photoreceptors harboring a defective gene in an animal model for a form of Leber Congenital Amaurosis known as LCA12. LCA is an inherited disease that causes severe vision loss in infants and is responsible for approximately 5% of all inherited retinal degenerative diseases. This study is important in further demonstrating the potential for gene therapy as a treatment for retinal degenerative diseases that are a leading cause of blindness in the world population. We believe this critical breakthrough will advance our understanding of LCA and lead to future treatment for this disorder. At MVRF, we are thrilled when the projects we fund make a substantial scientific contribution to help improve the lives of the millions of individuals affected by retinal diseases. The results of this study were published in the journal Human Molecular Genetics.
