Artificial Virus Delivers CRISPR-Cas9 System for Genome Editing of Cells in Mice

Citations Over TimeTop 1% of 2016 papers

Abstract

CRISPR-Cas9 has emerged as a versatile genome-editing platform. However, due to the large size of the commonly used CRISPR-Cas9 system, its effective delivery has been a challenge and limits its utility for basic research and therapeutic applications. Herein, a multifunctional nucleus-targeting "core-shell" artificial virus (RRPHC) was constructed for the delivery of CRISPR-Cas9 system. The artificial virus could efficiently load with the CRISPR-Cas9 system, accelerate the endosomal escape, and promote the penetration into the nucleus without additional nuclear-localization signal, thus enabling targeted gene disruption. Notably, the artificial virus is more efficient than SuperFect, Lipofectamine 2000, and Lipofectamine 3000. When loaded with a CRISPR-Cas9 plasmid, it induced higher targeted gene disruption efficacy than that of Lipofectamine 3000. Furthermore, the artificial virus effectively targets the ovarian cancer via dual-receptor-mediated endocytosis and had minimum side effects. When loaded with the Cas9-hMTH1 system targeting MTH1 gene, RRPHC showed effective disruption of MTH1 in vivo. This strategy could be adapted for delivering CRISPR-Cas9 plasmid or other functional nucleic acids in vivo.

Related Papers

• → CRISPR-Cas9 system: A new-fangled dawn in gene editing(2019)310 cited
• → Temperature effect on CRISPR-Cas9 mediated genome editing(2017)108 cited
• → Therapeutic applications of CRISPR RNA-guided genome editing(2016)25 cited
• → Elevated expression of exogenous RAD51 enhances the CRISPR/Cas9-mediated genome editing efficiency(2023)10 cited
• → CRISPR/Cas9 system: A promising technology for the treatment of inherited and neoplastic hematological diseases(2018)17 cited