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US 11,236,402 B2Granted
Adeno-associated virus virions with variant capsid
AssigneeThe Regents of the University of California
InventorsDavid V. Schaffer, Ryan R. Klimczak, James T. Koerber, John G. Flannery, Deniz Dalkara Mourot, Meike Visel, Leah C. T. Byrne
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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, when administered via intravitreal injection, 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.
Claims8
A recombinant adeno-associated virus 2 (rAAV2) virion comprising:
- a) a variant AAV capsid protein, wherein the variant AAV capsid protein comprises a peptide insertion relative to a corresponding parental AAV capsid protein as set forth in SEQ ID NO:
- 1, wherein the peptide insertion is between amino acids 587 and 588 relative to SEQ ID NO:
- 1, and wherein the peptide insertion comprises the amino acid LALGETTRPA (SEQ ID NO:
- 45);
- and
- b) a heterologous nucleic acid comprising a nucleotide sequence encoding an anti-angiogenic polypeptide.
3 more elements
Dependencies are inferred from the claim text.