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US 2019/0055581 A1Published application
Adeno-associated virus vector delivery of 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 for recombinant AAV vectors comprising a miniaturized human micro-dystrophin gene and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.
Claims24
A recombinant AAV vector comprising
- a) a nucleotide sequence having at least 85% identity to the nucleotide sequence SEQ ID NO:
- 7 and encodes a functional micro-dystrophin protein,
- b) the nucleotide sequence of SEQ ID NO:
- 7, or
- c) the nucleotide sequence of SEQ ID NO:
- 9.
3 more elements
Dependencies are inferred from the claim text.