Figure 5.

Nonviral delivery systems for in vivo genome editing via intramuscular injection. (A) Schematic representation of CRISPR-Gold synthesis. Intramuscular injection of CRISPR–Gold in mdx mice promoted homology-directed repair efficacy in the dystrophin gene, enhanced dystrophin protein expression, and reduced muscle fibrosis. Reproduced with permission from ref 78. Copyright 2017 Springer Nature. (B) A schematic illustration of the in situ free-radical polymerization of biodegradable nanocapsules (NCs) for the delivery of the Cas9 ribonucleoprotein (RNP) complex and its internalization and the subcellular release of the RNP. Local injections of NCs into the tibialis anterior muscle induced robust gene editing and strong tdTomato signals were observed. Reproduced with permission from ref 79. Copyright 2019 Springer Nature.