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Article

Genome editing in the mouse brain with minimally immunogenic Cas9 RNPs

4 Jul 2023

Abstract excerpt

Transient delivery of CRISPR-Cas9 ribonucleoproteins (RNPs) into the central nervous system (CNS) for therapeutic genome editing could avoid limitations of viral vector-based delivery including cargo capacity, immunogenicity, and cost. Here, we tested the ability of cell-penetrant Cas9 RNPs to edit the mouse striatum when introduced using a convection-enhanced delivery system. These transient Cas9 RNPs showed...

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Identifiers

PMID: 37403358
DOI: 10.1016/j.ymthe.2023.06.019

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