Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
AssigneeResearch Institute At Nationwide Children's Hospital
InventorsLouise Rodino-Klapac, Jerry R. Mendell
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Abstract
The invention provides gene therapy vectors, such as adeno-associated vims (AAV) vectors, expressing a miniaturized human micro-dystrophin gene and method of using these vectors to express micro-dystrophin in skeletal muscle including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.
Claims16
A method of treating muscular dystrophy comprising administering i) a therapeutically effective amount of a recombinant AAV vector comprising nucleotide 236 to nucleotide 4842 of SEQ ID NO:
- 3 and ii) a therapeutically effective amount of a recombinant AAV vector comprising nucleotides 120 to 2091 of SEQ ID NO:
- 6.
A method of increasing muscular force or muscle mass in a subject suffering from muscular dystrophy comprising administering i) a therapeutically effective amount of a recombinant AAV comprising nucleotide 236 to nucleotide 4842 of SEQ ID NO:
- 3 and ii) a therapeutically effective amount of a recombinant AAV vector comprising nucleotides 120 to 2091 of SEQ ID NO:
- 6.
A method of reducing or preventing fibrosis in a subject suffering from muscular dystrophy comprising administering i) a therapeutically effective amount of i) a recombinant AAV vector expressing comprising nucleotide 236 to nucleotide 4842 of SEQ ID NO:
- 3 and ii) a therapeutically effective amount of a recombinant AAV vector comprising nucleotides 120 to 2091 of SEQ ID NO:
- 6.
Dependencies are inferred from the claim text.