Dr. Shannon Boye, Assistant Professor in the University of Florida’s Department of Ophthalmology was recently awarded a $900,000 grant from Genzyme, a division of Sanofi, to develop a treatment for a severe form of childhood blindness called Leber congenital amaurosis-1 (LCA1). This disease is caused by genetic mutations which prevent the retina from responding to light. Patients are typically diagnosed within the first few months of life and remain profoundly visually impaired. Dr. Boye’s research shows that replacement of the gene using a viral vector restores function to the retina and results in long term useful vision in an otherwise blind mouse. Work is now underway to carry out pre-clinical studies required for FDA approval to test this therapy in Phase I clinical trials. This award supplements current funding from the Foundation Fighting Blindness (FFB) to Drs. William Hauswirth, PI and Shannon Boye, Co-PI and their clinical colleagues, Drs. Samuel Jacobson and Artur Cideyican. This exciting development highlights the Department of Ophthalmology’s continued dedication to the discovery of treatments for inherited retinal disease and further solidifies the University of Florida’s strong foothold in the field of gene therapy.
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