期刊
NATURE BIOTECHNOLOGY
卷 34, 期 2, 页码 204-+出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/nbt.3440
关键词
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资金
- Hereditary Disease Foundation
- Caltech-City of Hope Biomedical Initiative
- National Institutes of Health (NIH) Director's New Innovator [1DP2NS087949]
- NIH/National Institute on Aging (NIA) [1R01AG047664]
- Beckman Institute for CLARITY, Optogenetics and Vector Engineering Research
- Gordon and Betty Moore Foundation [GBMF2809]
- Caltech Programmable Molecular Technology Initiative
- NIH BRAIN [1U01NS090577]
- NIH/National Institute of Mental Health (NIMH) [1R21MH103824-01]
- Pew Charitable Trust
- Sloan Foundation
- Kimmel Foundation
- Human Frontiers in Science Program
- Caltech-GIST
- NIMH [1R01MH100900, 1R01MH100900-02S1]
- NIMH BRAINS [R01MH107800]
- California Institute of Regenerative Medicine (CIRM)
- MQ Fellow Award
- Donald E. and Delia B. Baxter Foundation Scholar Award
Recombinant adeno-associated viruses (rAAVs) are commonly used vehicles for in vivo gene transfer(1-6). However, the tropism repertoire of naturally occurring AAVs is limited, prompting a search for novel AAV capsids with desired characteristics(7-13). Here we describe a capsid selection method, called Cre recombination-based AAV targeted evolution (CREATE), that enables the development of AAV capsids that more efficiently transduce defined Cre-expressing cell populations in vivo. We use CREATE to generate AAV variants that efficiently and widely transduce the adult mouse central nervous system (CNS) after intravenous injection. One variant, AAV-PHP.B, transfers genes throughout the CNS with an efficiency that is at least 40-fold greater than that of the current standard, AAV9 (refs. 14-17), and transduces the majority of astrocytes and neurons across multiple CNS regions. In vitro, it transduces human neurons and astrocytes more efficiently than does AAV9, demonstrating the potential of CREATE to produce customized AAV vectors for biomedical applications.
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