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US 11,905,523 B2Granted
Adeno-associated viral vectors for treatment of Niemann-Pick Disease type-C
AssigneeGinkgo Bioworks, Inc.
InventorsDaniel McCoy, Garrett E. Berry, David Dismuke
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Abstract
Provided herein are gene therapy compositions and methods for treating, preventing, and/or curing NPC1. More specifically, the disclosure provides Adeno-associated virus (AAV) vectors for delivery of nucleic acids and nucleic acids (including AAV transfer cassettes) for treating, preventing, and/or curing NPC1.
Claims20
An adeno-associated virus (AAV) vector comprising:
- (i) a protein capsid comprising a capsid protein subunit comprising the sequence of SEQ ID NO:
- 180;
- and
- (ii) a nucleic acid encapsidated by the protein capsid;
- wherein the nucleic acid comprises a transfer cassette;
- wherein the transfer cassette comprises, from 5′ to 3′:
- a 5′ inverted terminal repeat (ITR);
- a promoter;
- a transgene that encodes a Niemann-Pick intracellular cholesterol transporter 1 (NPC1) protein;
- a polyadenylation signal;
- and
- a 3′ ITR.
9 more elements
Dependencies are inferred from the claim text.