Sanford L. Boye, M.S.
(352) 273-8805 (office)
(352) 392-3062 (fax)
I am a member of a highly collaborative team of investigators whose mission is focused on developing gene based therapies for blindness. My primary areas of research are 1) the design, construction and utilization of recombinant adeno-associated virus (AAV) vectors for gene transfer to the eye and 2) evaluation of efficacy, biodistribution and toxicity of clinically relevant AAV vectors in animal models of retinal disease. I have designed AAV vectors that have been used to successfully address a variety of retinal disease models, including autosomal recessive retinitis pigmentosa, achromatopsia, X-linked juvenile retinoschisis, Bardet-Biedl syndrome and multiple forms of Leber congenital amaurosis (LCA). I was a member of the National Eye Institute- funded consortium who developed an AAV gene therapy for the RPE65 version of Leber Congenital Amaurosis (LCA2). This led to several successful, first in human, clinical trials of gene therapy for LCA2. Currently I am working with Dr. Shannon Boye on both basic science- enhancing the AAV capsid for improved retinal transduction, and translational science- gaining FDA approval for an AAV- based treatment for the GUCY2D form of LCA (LCA1). I continue to collaborate closely with Dr. William Hauswirth on multiple projects including carrying on his work in the AAV vector production laboratory that he established.
- “Retinoschisin Mouse Model Re-derivation and Efficacy Testing of AAV-RS1” (SL Boye)
Funding Agency: AGTC
- “Evaluation of rAAV2tYF-CB-hRS1 Tox/BD Drug product for Transduction Efficiency and Rescue of ERG Responses in RS1 Knock-out Mice” (SL Boye and Hauswirth)
Funding Agency: AGTC
- “Toxicology, Biodistribution and Efficacy Study of rAAV2tYF-CB-hRS1” (subproject- SL Boye)
Funding Agency: Applied Genetics Technologies Corporation (AGTC)
- “Gene Therapy for LCA1” (SE Boye)
Funding Agency: Genzyme-Sanofi
- “Vision Research Core” (P30EY021721)
Funding Agency: NIH/National Eye Institute
Role: Senior personnel, modules supervisor, (PI, Hauswirth)