Biology

Engineering self-deliverable ribonucleoproteins for genome editing in the brain

K. Chen, E. C. Stahl, et al.

This groundbreaking research conducted by Kai Chen, Elizabeth C. Stahl, Min Hyung Kang, Bryant Xu, Ryan Allen, Marena Trinidad, and Jennifer A. Doudna introduces innovative self-deliverable CRISPR ribonucleoproteins that enhance genome editing in the brain. By engineering Cas9 proteins with effective cell-penetrating peptides, this study significantly improves editing efficacy in neural progenitor cells and demonstrates robust genome editing capabilities in live mice, paving the way for advanced brain therapies.

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Abstract

Effective delivery of CRISPR ribonucleoproteins (RNPs) for genome editing remains challenging, particularly in the brain. This study engineers self-deliverable RNPs by fusing cell-penetrating peptides (CPPs) to the Cas9 protein. Screening identified A22p, a peptide derived from human semaphorin-3a, as particularly effective. Engineered Cas9 RNPs with three copies of A22p demonstrated significantly improved editing efficacy in neural progenitor cells *in vitro* and robust genome editing in the mouse striatum *in vivo*, offering a facile platform for genome editing in the brain.

Publisher

Nature Communications

Published On

Feb 26, 2024

Authors

Kai Chen, Elizabeth C. Stahl, Min Hyung Kang, Bryant Xu, Ryan Allen, Marena Trinidad, Jennifer A. Doudna

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