Compositions and methods for muscle disorders
AssigneeKate Therapeutics, Inc.
InventorsMark Fielden, Mohammadsharif Tabebordbar, Jana Jenquin, Brian Spencer
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Abstract
The invention discloses approaches to reduce expression of a gene in a cell.
Claims18
An adeno-associated virus (AAV) vector comprising:
- a promoter sequence,
- a miRNA strand that binds to DUX4-FL, and
- a capsid protein comprising at least one modification that is an insertion between any two contiguous amino acids between amino acids 262-269, 327-332, 382-386, 452-460, 488-505, 527-539, 545-558, 581-593, 704-714, or any combination thereof in an AAV9 capsid polypeptide or in an analogous position in an AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV rh.74, AAV rh.10 capsid polypeptide.
A method of inhibiting expression of a gene in a cell, the method comprising administering to a subject an adeno-associated virus (AAV) vector comprising a promoter sequence, a miRNA strand that binds to DUX4-FL, and a capsid protein comprising at least one modification that is an insertion between any two contiguous amino acids between amino acids 262-269, 327-332, 382-386, 452-460, 488-505, 527-539, 545-558, 581-593, 704-714, or any combination thereof in an AAV9) capsid polypeptide or in an analogous position in an AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, AAV8, AAV rh.74, AAV rh.10 capsid polypeptide.
Dependencies are inferred from the claim text.