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US 12,611,436 B2Granted
AAV transfer cassette
AssigneeSarepta Therapeutics, Inc.
InventorDavid Dismuke
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Abstract
Described herein are AAV transfer cassettes and plasmids used in the production of recombinant adeno-associated viral (rAAV) vectors. The disclosed cassettes and plasmids comprise one or more transgenes having therapeutic efficacy in the amelioration, treatment and/or prevention of one or more diseases or disorders, such as Niemann-Pick Disease, type C1 (NPC1).
Claims4
A method for treating a subject having Neimann-Pick Disease Type C1 (NPCI) in need thereof, comprising:
- systemically administering to the subject a recombinant AAV vector comprising a protein capsid and a nucleic acid encapsidated by the protein capsid,
- wherein the nucleic acid comprises a transfer cassette comprising the sequence of SEQ ID NO:
- 14 and the protein capsid comprises a capsid protein subunit from an AAV9 serotype,
- and
- wherein treatment reduces cholesterol accumulation in cells of the subject transduced by the recombinant AAV vector.
2 more elements
A method for treating a subject having Neimann-Pick Disease Type C1 (NPCI) in need thereof, comprising:
- systemically administering to the subject a recombinant AAV vector comprising a protein capsid and a nucleic acid encapsidated by the protein capsid,
- wherein the nucleic acid comprises a transfer cassette comprising the sequence of any one of SEQ ID NOS:
- 15-19 and the protein capsid comprises a capsid protein subunit from an AAV9 serotype,
- and
- wherein treatment reduces cholesterol accumulation in cells of the subject transduced by the recombinant AAV vector.
2 more elements
Dependencies are inferred from the claim text.