Gene therapy for ocular disorders
AssigneeThe Trustees of the University of Pennsylvania
InventorsJean Bennett, Jeannette Bennicelli, Junwei Sun
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Abstract
Compositions and methods are provided for treating ocular disorders in a subject are provided. In one aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGA3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGB3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding REP-1. In desired embodiments, the subject is human, cat, dog, sheep, or non-human primate.
Claims10
A method for treating achromatopsia in a human subject in need thereof, said method comprising administering to the human subject subretinally an AAV vector comprising an AAV capsid and a nucleic acid sequence packaged therein, wherein the nucleic acid sequence comprises i) AAV ITR sequences, ii) a nucleic acid sequence encoding human cyclic nucleotide gated channel alpha 3 (CNGA3), and iii) expression control sequences that direct expression of the CNGA3 in a host cell, wherein the nucleic acid sequence encoding the human CNGA3 sequence comprises SEQ ID NO:
- 9 or SEQ ID NO:
- 11.
Dependencies are inferred from the claim text.