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

Autor: Stephen N. Floor, Anirvan Ghosh, Claire Coulon-Bainier, Ashwin A Banfal, Brett T. Staahl, Jennifer K Sabo, Gabriela Acevedo Munares, Madhurima Benekareddy, Cole Urnes, Jennifer A. Doudna
Rok vydání: 2017
Předmět:
0301 basic medicine
Male
1.1 Normal biological development and functioning
CRISPR-Associated Proteins
Biomedical Engineering
Hippocampus
Bioengineering
Nerve Tissue Proteins
Striatum
Biology
Protein Engineering
Applied Microbiology and Biotechnology
Article
03 medical and health sciences
Mice
0302 clinical medicine
Genome editing
Bacterial Proteins
CRISPR-Associated Protein 9
MD Multidisciplinary
Genetics
Animals
Clustered Regularly Interspaced Short Palindromic Repeats
Ribonucleoprotein
Gene Editing
Cas9
Targeted Gene Repair
Human Genome
Neurosciences
Brain
Endonucleases
Brain Disorders
Cell biology
Cortex (botany)
030104 developmental biology
nervous system
Ribonucleoproteins
Neurological
Gene Targeting
Molecular Medicine
030217 neurology & neurosurgery
Nuclear localization sequence
Biotechnology
Zdroj: Nature biotechnology, vol 35, iss 5
Popis: 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.
Databáze: OpenAIRE
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