Gene Therapy for BEST1 Dominant Mutations
AssigneesUniversity of Rochester, The Trustees of Columbia University in the City of New York
InventorsTingting Yang, Yu Zhang, Stephen Tsang
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Abstract
The present invention provides compositions and methods for treatment of bestrophinopathies. In certain embodiments, the invention treats, improves retinal function, and prevents progression of disorder in a subject with retinal degenerative disorder. The present invention comprises administration of a composition comprising wild-type BEST1 gene to subjects with a BEST1 mutation, in need of improved retinal function.
Claims21
A method of treating a retinal degenerative disorder associated with a BEST1 dominant mutation in a subject, the method comprising administering to a subject in need thereof an effective amount of a composition comprising a nucleic acid molecule encoding wild-type BEST1.
A cell having an endogenous BEST1 dominant mutation comprising an exogenous nucleic acid molecule that encodes wild-type BEST1.
Dependencies are inferred from the claim text.