CRISPR/Cas9 Nano-delivery Approaches for Targeted Gene Therapy

Abstract

Gene-/genome-editing technology achieved its pinnacle of success in its therapeutic applications with the discovery of the CRISPR/Cas9 system. The CRISPR system is the short form for clustered regularly interspersed short palindromic repeats that exist in bacteria and archaea as a part of their adaptive immune system to fight against plasmids and other viral genetic elements. The potential of this natural editing mechanism to alter faulty genes in a spectrum of human diseases has been groundbreaking in various aspects. However, the safe and effective delivery of the CRISPR/Cas9 system to the target cells has become a challenge and topic of discussion in recent years. This chapter discusses the current scenario of effective delivery strategies, and challenging aspects, in addition, to highlighting the present trends of incorporating nanoparticle-mediated platforms such as polymeric nanoparticles, liposomes, lipid nanoparticles, etc. that will assist in the future clinical translation of CRISPR/Cas9 system for therapeutic purposes.