Tissue-targeted modified aav capsids and methods of use thereof
AssigneeLocanabio, Inc.
InventorGreg NACHTRAB
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Abstract
Provided herein are modified AAV capsids comprising modified AAV capsid proteins having improved transduction in muscle tissue, reduced immunogenicity, reduced neutralizing antibody binding, reduced liver tissue transduction, and combinations thereof. Also provided are methods of treating a patient with AAV viral vectors comprising modified AAV capsid proteins.
Claims41
A modified AAV capsid protein comprising a modified variable region (VR) VIII.
The modified AAV capsid protein of any one of the preceding claims, wherein the capsid protein comprises an amino acid sequence at least 85%, 90%, 95%, 96%, 97%, 98%, 99% or 100% identical to the amino acid sequence set forth in any one of SEQ ID NOs:
- 53, 75, 102, 124, 245, 249, 255, 258, 262, or 267.
A modified AAV capsid protein comprising an amino acid sequence at least 85%, 90%, 95%, 96%, 97%, 98%, 99% or 100% identical to the amino acid sequence set forth in any one of SEQ ID NOs:
- 53, 75, 102, 124, 245, 249, 255, 258, 262, or 267.
Dependencies are inferred from the claim text.