Non-viral CRISPR/Cas gene editing in vitro and in vivo enabled by co-delivery of mRNA and sgRNA inside of #synthetic lipid nanoparticles

摘要

Conclusions: In summary, we will report the development of the first non-viral delivery system for in vitro and in vivo co-delivery of Cas9 mRNA and targeted sgRNA. We quantified the kinetic events for CRISPR/Cas editing, and defined the timing for optimal delivery. Editing was confirmed by Surveyor, and was permanent. Finally, we will show that i.v. co-delivery can be achieved in vivo. Cumulatively, these results show that CRISPR/Cas editing is possible using non-viral carriers. The use of scalable and translatable technologies, such as ZNPs, will provide powerful tools for in vivo gene editing to understand biology, create animal models, and treat diseases.