Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof
AssigneeGenzyme Corporation
InventorsAmy Frederick, Xiaoying Jin, Lin Liu, Catherine O'Riordan, Jennifer Sullivan
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Abstract
Provided herein are modified adeno-associated viral (AAV) capsid proteins, compositions (e.g., rAAV) comprising the capsid proteins, and nucleic acids encoding the capsid proteins. The AAV capsids provided herein confer retinal cell tropism and/or corneal cell tropism, and mediate improved transduction efficiency in clinically relevant ocular cell types such as photoreceptors and/or corneal endothelial cells. Also provided are nucleic acids encoding the capsid proteins, and AAV particles comprising the capsid proteins.
Claims28
A modified adeno-associated virus 5 (AAV5) capsid protein comprising the amino acid sequence set forth in SEQ ID NOs:
- 3, 5, 7, or 9.
A modified AAV5 capsid protein comprising:
- a G at the position corresponding to amino acid 194;
- an R at the position corresponding to amino acid 564;
- or
- an R at the position corresponding to amino acid 573;
- wherein the modified AAV5 capsid protein is modified from a wild-type AAV5 capsid protein;
- and
- wherein numbering of the position is based on the amino acid sequence of the wild-type AAV5 VP1 as set forth in SEQ ID NO:1.
Dependencies are inferred from the claim text.