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US 2020/0046851 A1Published application
Gene therapy for retinitis pigmentosa
AssigneeGenzyme Corporation
InventorsCatherine O'RIORDAN, Matthew ADAMOWICZ
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Abstract
Provided herein are methods for treating retinitis pigmentosa using an AAV particles encoding miR-708. In one aspect, viral particles are administered to the eye of a human subject; for example, by subretinal injection. Viral particles comprising AAV5 capsids or mutants thereof are contemplated.
Claims44
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A rAAV particle comprising a rAAV vector comprising a nucleic acid encoding a miR-708.
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Dependencies are inferred from the claim text.