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Lookup NU author(s): Dr Abhishek Banerjee
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© 2015 Nature America, Inc. All rights reserved.Probing gene function in the mammalian brain can be greatly assisted with methods to manipulate the genome of neurons in vivo. The clustered, regularly interspaced, short palindromic repeats (CRISPR)-associated endonuclease (Cas)9 from Streptococcus pyogenes (SpCas9) can be used to edit single or multiple genes in replicating eukaryotic cells, resulting in frame-shifting insertion/deletion (indel) mutations and subsequent protein depletion. Here, we delivered SpCas9 and guide RNAs using adeno-associated viral (AAV) vectors to target single (Mecp2) as well as multiple genes (Dnmt1, Dnmt3a and Dnmt3b) in the adult mouse brain in vivo. We characterized the effects of genome modifications in postmitotic neurons using biochemical, genetic, electrophysiological and behavioral readouts. Our results demonstrate that AAV-mediated SpCas9 genome editing can enable reverse genetic studies of gene function in the brain.
Author(s): Swiech L, Heidenreich M, Banerjee A, Habib N, Li Y, Trombetta J, Sur M, Zhang F
Publication type: Article
Publication status: Published
Journal: Nature Biotechnology
Year: 2015
Volume: 33
Issue: 1
Pages: 102-106
Online publication date: 19/10/2014
Acceptance date: 02/10/2014
ISSN (print): 1087-0156
ISSN (electronic): 1546-1696
Publisher: Nature Publishing Group
URL: https://doi.org/10.1038/nbt.3055
DOI: 10.1038/nbt.3055
PubMed id: 25326897
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