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US 2026/0131032 A1Published application
Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy
AssigneeResearch Institute At Nationwide Children's Hospital
InventorsLouise Rodino-Klapac, Jerry R. Mendell
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Abstract
Described herein are methods of treating muscular dystrophy comprising administering a recombinant AAV (rAAV) scAAVrh74.MHCK7.hSGCB vector, methods of expressing beta-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV, and methods of generating the rAAV.
Claims21
A polynucleotide comprising a nucleotide sequence that is at least 90%, 95%, or 99% identical to the nucleotide sequence of SEQ ID NO:
- 24.
A polynucleotide comprising a nucleotide sequence identical to the sequence of SEQ ID NO:
- 19.
Dependencies are inferred from the claim text.