Invention Application
- Patent Title: AAV CAPSIDS VARIANTS AND USES THEREOF
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Application No.: US17915548Application Date: 2021-03-30
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Publication No.: US20230138766A1Publication Date: 2023-05-04
- Inventor: Guangping Gao , Guangchao Xu , Phillip Tai , Yuquan Wei , Li Luo
- Applicant: University of Massachusetts , Sichuan University
- Applicant Address: US MA Boston; CN Chengdu
- Assignee: University of Massachusetts,Sichuan University
- Current Assignee: University of Massachusetts,Sichuan University
- Current Assignee Address: US MA Boston; CN Chengdu
- International Application: PCT/US2021/024837 WO 20210330
- Main IPC: C12N15/86
- IPC: C12N15/86 ; A61K9/00 ; C07K14/005

Abstract:
Aspects of the disclosure relate to compositions and methods for delivering a transgene (e.g., a transgene encoding one or more gene products) to a target cell. The disclosure is based, in part, on adeno-associated virus (AAV) capsid protein variants characterized by tropisms for certain cell types (e.g., neurons, muscle cells, bone cells, heart cells, etc.). In some embodiments, recombinant A A Vs (rAAVs) comprising the capsid protein variants (e.g., AAVv66, SEQ ID NO: 1) are more efficiently packaged than rAAVs having certain wild-type AAV capsid proteins. Methods of delivering an rAAV comprising the AAV capsid protein variants are also described by the disclosure.
Information query
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