Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
AssigneeNationwide Children's Hospital, Inc.
InventorsJerry R. Mendell, Louis Chicoine, Louise Rodino-Klapac, Kelly Reed Clark, Thomas J. Preston
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Abstract
The present invention relates to recombinant adeno-associated virus (rAAV) delivery of an alpha-sarcoglycan gene. The invention provides rAAV products and methods of using the rAAV in the treatment of limb girdle muscular dystrophies such as LGMD2D.
Claims5
A method of improving muscle function in a patient afflicted with limb girdle muscular dystrophy type 2D (LGMD 2D) comprising the step of administering the patient with an encapsidated recombinant adeno-associated (AAV) comprising an AAVrh.74 capsid that comprises the amino acid sequence of SEQ ID NO:
- 2;
- wherein
- the genome of the recombinant AAV comprises a gene expression cassette comprising the polynucleotide of SEQ ID NO:
- 3 encoding an alpha-sarcoglycan under the transcriptional control of a promoter, said cassette flanked by one or more AAV inverted terminal repeats.
Dependencies are inferred from the claim text.