期刊
NATURE BIOTECHNOLOGY
卷 35, 期 5, 页码 431-+出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/nbt.3806
关键词
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资金
- F. Hoffmann-La Roche Postdoctoral Fellowship [RPF311]
- Roche Pharmaceutical's Roche Alliance with Distinguished Scientists (ROADS) Fund
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.
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