Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 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 recombinant AAV vectors comprising a polynucleotide sequence comprising β-sarcoglycan and methods of using the recombinant vectors to reduce or prevent fibrosis in a mammalian subject suffering from a muscular dystrophy. Also described herein are combination therapies comprising administering AAV vectors(s) expressing β-sarcoglycan and miR-29c to a mammalian subject suffering from a muscular dystrophy.
Claims17
1-49. (canceled)
A plasmid comprising a polynucleotide sequence that comprises a nucleotide sequence at least 95% identical to SEQ ID NO:
- 1, wherein said plasmid lacks AAV rep and cap genes.
A method of producing a recombinant AAV (rAAV) particle comprising transfecting a cell with a plasmid,
- wherein the plasmid comprises a nucleotide sequence at least 95% identical to SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.