Efficient systemic treatment of dystrophic muscle pathologies
AssigneesGenethon, Royal Holloway and Bedford New College
InventorsGeorge Dickson, Thomas Voit, Philippe Moullier, Caroline Le Guiner
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Abstract
A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein: the gene therapy product comprises a nucleic acid sequence encoding a functional microdystrophin; the composition is systemically administered.
Claims7
1-16. (canceled)
A gene therapy product, comprising an adeno-associated viral (AAV) vector which harbors a nucleic acid sequence encoding a human ΔR4-R23/ΔCT microdystrophin, wherein the AAV vector is an AAV2/8 vector, and wherein the nucleic acid sequence comprises SEQ ID NO:
- 1.
A pharmaceutical composition, comprising a gene therapy product and a pharmaceutically acceptable carrier, wherein the gene therapy product comprises an adeno-associated viral (AAV) vector which harbors a nucleic acid sequence encoding a human ΔR4-R23/ΔCT microdystrophin, and wherein the AAV vector is an AAV2/8 vector, and wherein the nucleic acid sequence comprises SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.