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AssigneeThe Trustees of the University of Pennsylvania
InventorsJean Bennett, Jeannette Bennicelli, Junwei Sun
Estimating the term…
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.
An adeno-associated virus (AAV) vector comprising an AAV capsid and a nucleic acid sequence comprising AAV inverted terminal repeat (ITR) sequences and a nucleic acid sequence encoding human Rab Escort Protein-1 (REP-1), and expression control sequences that direct expression of the REP-1 in a host cell.
A method for treating achromatopsia, said method comprising administering to a subject in need thereof an AAV vector comprising an AAV capsid and a nucleic acid sequence comprising AAV ITR sequences and a nucleic acid sequence encoding human cyclic nucleotide gated channel alpha 3 (CNGA3), and expression control sequences that direct expression of the CNGA3 in a host cell.
A viral vector comprising a vector genome comprising a 5′ ITR, a CMV enhancer, a Chicken beta-actin promoter, CBA exon 1 and intron, a Kozak sequence, a nucleic acid sequence encoding human Rab Escort Protein-1 (REP-1), bGH poly A, and a 3′ ITR.
Dependencies are inferred from the claim text.