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US 9,457,103 B2Granted
Adeno-associated virus virions with variant capsid and methods of use thereof
AssigneeThe Regents of the University of California
InventorsDavid V. Schaffer, Ryan R. Klimczak, James T. Koerber, John G. Flannery
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Abstract
The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells compared to wild-type AAV. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.
Claims27
A recombinant adeno-associated virus (rAAV) virion comprising:
- a) a variant AAV capsid protein, wherein the variant AAV capsid protein comprises an amino acid substitution at amino acid 532 of the AAV6 capsid sequence as set forth in SEQ ID NO:1, or the corresponding position in another AAV parental serotype, and wherein the variant capsid protein confers increased infectivity of a retinal cell compared to the infectivity of the retinal cell by an AAV virion comprising a wild-type AAV capsid protein, wherein the AAV capsid protein does not comprise an amino acid sequence present in a naturally occurring AAV capsid protein;
- and
- b) a heterologous nucleic acid comprising a nucleotide sequence encoding a gene product.
Dependencies are inferred from the claim text.