Recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
AssigneeThe Board of Trustees of the Leland Stanford Junior University
InventorsMark A. Kay, Nicole K. Paulk
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Abstract
The present invention relates to variant AAV capsid polypeptides, wherein the variant AAV capsid polypeptides exhibit increased transduction and/or tropism in human muscle tissue or cells as compared non-variant parent capsid polypeptides.
Claims41
A variant adeno-associated virus (AAV) capsid polypeptide, wherein said variant AAV capsid polypeptide exhibits increased transduction or tropism in human skeletal muscle tissue or cells as compared to a non-variant parent capsid polypeptide, wherein said variant AAV capsid polypeptide comprises a sequence selected from the group consisting of AAV-NP6 (SEQ ID NO:
- 1), AAV-NP20 (SEQ ID NO:
- 2), AAV-NP22(SEQ ID NO:
- 3), AAV-NP36 (SEQ ID NO:
- 4), AAV-NP66 (SEQ ID NO:
- 5), AAV-NP81 (SEQ ID NO:
- 6), and AAV-NP94 (SEQ ID NO:
- 7).
A variant adeno-associated virus (AAV) capsid polypeptide, wherein said variant AAV capsid polypeptide exhibits enhanced neutralization profile as compared to a non-variant parent capsid polypeptide, wherein said variant AAV capsid polypeptide comprises a sequence selected from the group consisting of AAV-NP6 (SEQ ID NO:
- 1), AAV-NP20 (SEQ ID NO:
- 2), AAV-NP22 (SEQ ID NO:
- 3), AAV-NP36 (SEQ ID NO:
- 4), AAV-NP66 (SEQ ID NO:
- 5), AAV-NP81 (SEQ ID NO:
- 6), and AAV-NP94 (SEQ ID NO:
- 7).
Dependencies are inferred from the claim text.