Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems

Ken Y. Chan, Min J. Jang, Bryan B. Yoo, Alon Greenbaum, Namita Ravi, Wei Li Wu, Luis Sánchez-Guardado, Carlos Lois, Sarkis K. Mazmanian, Benjamin E. Deverman, Viviana Gradinaru

研究成果: Article同行評審

770 引文 斯高帕斯(Scopus)

摘要

Adeno-associated viruses (AAVs) are commonly used for in vivo gene transfer. Nevertheless, AAVs that provide efficient transduction across specific organs or cell populations are needed. Here, we describe AAV-PHP.eB and AAV-PHP.S, capsids that efficiently transduce the central and peripheral nervous systems, respectively. In the adult mouse, intravenous administration of 1 × 10 11 vector genomes (vg) of AAV-PHP.eB transduced 69% of cortical and 55% of striatal neurons, while 1 × 10 12 vg of AAV-PHP.S transduced 82% of dorsal root ganglion neurons, as well as cardiac and enteric neurons. The efficiency of these vectors facilitates robust cotransduction and stochastic, multicolor labeling for individual cell morphology studies. To support such efforts, we provide methods for labeling a tunable fraction of cells without compromising color diversity. Furthermore, when used with cell-type-specific promoters and enhancers, these AAVs enable efficient and targetable genetic modification of cells throughout the nervous system of transgenic and non-transgenic animals.

原文English
頁(從 - 到)1172-1179
頁數8
期刊Nature Neuroscience
20
發行號8
DOIs
出版狀態Published - 2017 8月 1

All Science Journal Classification (ASJC) codes

  • 一般神經科學

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