Dr. Hauswirth’s research team focuses on developing Adeno-associated virus (AAV) gene therapies for the treatment of ocular disease. His group specializes in developing and then testing these therapies in animal models of retinal degeneration. The gene vector platform, AAV, has been shown to be safe and effective in human clinical trials of Leber Congenital Amaurosis type 2 (LCA2) a form of childhood retinal disease. Dr. Hauswirth’s research was instrumental in the lead up to the LCA2 clinical trials. He and collaborators from the University of Pennsylvania undertook one of the ground breaking clinical trials. He and his team continue to develop AAV vectors with the goal of future clinical trials for other retinal degenerative diseases such as Retinitis Pigmentosa, Stargardt disease, Achromatopsia, Retinoschisis and Age-related Macular Degeneration (AMD).
For more information about Dr. Hauswirth’s lab please contact Dr. Sanford Boye at sboye@ufl.edu