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Nat Biotechnol. 2017 May;35(5):431-434. doi: 10.1038/nbt.3806. Epub 2017 Feb 13.

Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes.

Author information

1
Department of Molecular and Cell Biology, University of California, Berkeley, California, USA.
2
Roche Pharma Research and Early Development, Basel, Switzerland.
3
E-Scape Bio, San Francisco, California, USA.
4
Howard Hughes Medical Institute, University of California, Berkeley, California, USA.
5
Innovative Genomics Institute, University of California, Berkeley, California, USA.
6
MBIB Division, Lawrence Berkeley National Laboratory, Berkeley, California, USA.
7
Department of Chemistry, University of California, Berkeley, California, USA.

Abstract

We demonstrate editing of post-mitotic neurons in the adult mouse brain following injection of Cas9 ribonucleoprotein (RNP) complexes in the hippocampus, striatum and cortex. Engineered variants of Cas9 with multiple SV40 nuclear localization sequences enabled a tenfold increase in the efficiency of neuronal editing in vivo. These advances indicate the potential of genome editing in the brain to correct or inactivate the underlying genetic causes of neurological diseases.

PMID:
28191903
DOI:
10.1038/nbt.3806
[Indexed for MEDLINE]

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