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US 12,285,497 B2Granted
Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
AssigneeResearch Institute At Nationwide Children's Hospital
InventorsLouise Rodino-Klapac, Jerry R. Mendell
Life of this patent
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Abstract
Described herein are methods of treating muscular dystrophy comprising administering a self complementary recombinant AAV (rAAV) ScAAVrh74.MIHCK7.h8GCB vector, methods of expressing beta-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV, and methods of generating the rAAV.
Claims11
A polynucleotide sequence comprising a first nucleotide sequence at least 95% identical to the nucleotide sequence of SEQ ID NO:
- 1, wherein SEQ ID NO:
- 1 comprises a second nucleotide sequence encoding a β-sarcoglycan protein.
Dependencies are inferred from the claim text.