Gene therapy for limb-girdle muscular dystrophy type 2c
AssigneeResearch Institute At Nationwide Children's Hospital
InventorLouise R. RODINO-KLAPAC
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Abstract
The disclosure relates to gene therapy vectors, such as AAV vectors, comprising a polynucleotide encoding γ-sarcoglycan (SGCG) and methods of using such gene therapy vectors to treat subjects suffering from a muscular dystrophy. e.g. limb girdle dystrophy type 2C (LGMD2C).
Claims20
A method of treating γ-sarcoglycanopathy in a subject, comprising administering to the subject a therapeutically effective amount of a composition comprising a polynucleotide sequence encoding γ-sarcoglycan.
A polynucleotide comprising a nucleotide sequence encoding γ-sarcoglycan, wherein the nucleotide sequence comprises a nucleotide sequence at least 95% identical to SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.