Nanoparticles for CRISPR-Cas9 delivery

Abstract

The DNA mutation that causes Duchenne muscular dystrophy in mice can be corrected, with minimal off-target effects, by gold nanoparticles carrying the CRISPR components.

Fig. 1. Genome editing via nanoparticle-mediated delivery of the CRISPR–Cas9 machinery

a, The nanoparticles are composed of a gold core and a shell of endosomal-disrupting cationic polymer, with the Cas9–gRNA protein complex and the donor template DNA packaged in between. b, The Cas9–gRNA ribonucleoprotein generates a cut site at a particular location of the cell’s DNA, and the donor DNA molecule is used as a template to repair the cut site via homology-directed repair (HDR). DSB, double-strand break; gRNA, guide RNA. Figure adapted from: a, ref. , Macmillan Publishers Ltd; b, ref. , Macmillan Publishers Ltd.