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US 10,647,751 B2Granted
Production of large-sized microdystrophins in an AAV-based vector configuration
AssigneeRoyal Holloway and Bedford New College
InventorGeorge Dickson
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Abstract
An adeno-associated viral (AAV) vector containing an expression construct, wherein: the expression construct comprises a nucleic acid sequence which encodes a microdystrophin (MD); and the nucleic acid sequence encoding the MD has a size of at least 4.1 kb.
Claims19
An adeno-associated viral (AAV) vector comprising an expression construct, wherein:
- the expression construct comprises a nucleic acid sequence which encodes a microdystrophin (MD);
- the nucleic acid sequence encoding the MD has a size of at least 4.1 kb;
- the expression construct has a size of less than 6 kb;
- and
- the MD contains a partial or full-length C terminal domain of dystrophin.
2 more elements
An adeno-associated viral (AAV) vector comprising an expression construct, wherein:
- the expression construct comprises a nucleic acid sequence which encodes a microdystrophin (MD);
- the nucleic acid sequence encoding the MD has a size of at least 4.1 kb;
- the MD contains a partial or full length C terminal domain of dystrophin, and
- said MD comprises a central rod domain and has a deletion ΔR4-23 in the central rod domain and wherein the partial or full-length C terminal domain is encoded by exons 70 to 75 or exons 70 to 79, respectively.
1 more element
Dependencies are inferred from the claim text.