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US 12,653,909 B2Granted
Modified AAV capsids and uses thereof
AssigneeSolid Biosciences Inc.
InventorPeter Pechan
Life of this patent
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Abstract
The invention described herein provide a modified VPI capsid enabling preferential targeted expression of gene of interest (GOI) in muscle tissues, as well as recombinant adeno-associated virus (rAAV) with the GOI packaged with the modified VPI capsids, and uses thereof.
Claims21
A modified adeno-associated virus (mAAV) capsid polypeptide consisting of the polypeptide of SEQ ID NO:
- 14.
A modified adeno-associated virus (mAAV) capsid polypeptide comprising the polypeptide of SEQ ID NO:
- 14.
A recombinant adeno-associated virus (rAAV), comprising:
- a modified adeno-associated virus (mAAV) capsid polypeptide consisting of the polypeptide of SEQ ID NO:
- 14, wherein the VP1 capsid of the rAAV consists of or consists essentially of the mAAV capsid polypeptide;
- and
- a gene of interest (GOI) flanked by a pair of AAV inverted terminal repeat (ITR) sequences, wherein the GOI is a microdystrophin gene encoding a protein comprising, from N- to C-terminus, an amino-terminal actin-binding (AB1) domain, a ß-dystroglycan binding domain, a Hinge 1 domain (H1), a spectrin-like repeat domain consisting of five spectrin-like repeats that include spectrin-like repeat 1 (SR1), spectrin-like repeat 16 (SR16), spectrin-like repeat 17 (SR17), spectrin-like repeat 23 (SR23), and spectrin-like repeat 24 (SR24), and a Hinge 4 domain (H4), wherein the microdystrophin gene is operatively linked to a muscle-specific human muscle creatine kinase CK8 promoter, and wherein the GOI is flanked by a pair of AAV2 ITR sequences.
1 more element
Dependencies are inferred from the claim text.