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Abstract #2272

The Role of the Human Visual Cortex in Assessment of the Long-term Durability of Retinal Gene Therapy in Follow-on RPE65 Clinical Trial Patients

Manzar Ashtari1,2,3, Elena Nikonova4, Kathleen A. Marshall5, Gloria J. Young1, Puya Aravand1, Wei Pan6, Gui-shuang Ying6, Aimee E. Willett1, Mani Mahmoudian1, Albert M. Maguire1,2,5, and Jean Bennett1,2,5

1Center for Advanced Retinal and Ocular Therapeutics (CAROT), University of Pennsylvania, Philadelphia, PA, United States, 2F.M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA, United States, 3Department of Radiology, University of Pennsylvania, Philadelphia, PA, United States, 4University of Pittsburgh, Pittsburgh, PA, United States, 5Center for Cellular and Molecular Therapeutics, The Children’s Hospital of Philadelphia, Philadelphia, PA, United States, 6Westat Biostatistics and Data Management Core, University of Pennsylvania, Philadelphia, PA, United States

Leber’s congenital amaurosis (LCA) is a rare blinding disease with no cure. Recently, patients with LCA underwent retinal gene therapy and regained their vision to a great extent. We followed this group of LCA patients before and up to three years on an annual basis after gene therapy using fMRI to assess the feasibility and durability of retinal gene therapy over time and the role fMRI could play as an outcome measure for other future retinal interventions.

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