CRISPR/Cas9 system in hematopoietic stem cells: Basic research and clinical applications

Abstract

Clustered regularly interspaced short palindromic repeats (CRISPR) approach adapted from the prokaryotic adaptive immunity system against pathogen attack is so valuable and promising tool for treatment of human malignant and non-malignant hematological disease and disorders through gene editing in hematopoietic stem cells (HSCs). Moreover, the CRISPR/Cas9 approach is not only useful for therapeutic purposes; it is considerably preferred for the generation of in vitro and in vivo animal disease models. CRISPR/Cas9 approach has been developed for highly efficient on-target cleavage, and low off-target effect via delivery systems and manipulation of CRISPR components including single guide RNA (sgRNA) and Cas enzymes. In this review, we discussed the CRISPR/Cas9 system applications on hematopoietic stem cells in basic research and clinical area with basic research and clinical perspectives.