Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 28191903.
- Also identified by DOI 10.1038/nbt.3806 and PMC identifier 6649674.
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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.
Medical subject headings
- Bacterial Proteins
- Brain
- CRISPR-Associated Proteins
- Endonucleases
- Gene Editing
- Nerve Tissue Proteins
- Ribonucleoproteins