Methods and applications of CRISPR/Cas system for genome editing in stem cells

Abstract

Genome editing technology holds great promise for genome manipulation and gene therapy. While widespread utilization, genome editing has been used to unravel the roles of specific genes in differentiation and pluripotency of stem cells, and reinforce the stem cell-based applications. In this review, we summarize the advances of genome editing technology, as well as the derivative technologies from CRISPR/Cas system, which show tremendous potential in various fields. We also highlight the key findings in the studies of stem cells and regeneration by genome editing technology.

rAAV-Mediated Cochlear Gene Therapy: Prospects and Challenges for Clinical Application

“…HDR, on the other hand, performs a precise DNA repair using a dsDNA template provided with the short-guide RNA and a Cas-like enzyme. Therefore, this provides optional methods for the correction of genetic disorders [111,112]. rAAVs have mostly been the vector of choice for CRISPR genome editing [113].…”

Crispr - Cas9 Gene Editing: A Review

“…Derived from the inner cell mass (ICM) of blastocytes embryonic stem cells are Pluripotent stem cells (PSC) and hence shows difficulty in genome editing [46]. CRISPR -Cas system can efficiently control the genome without complex process [23,46]. This accelerates the stem cell study.…”

“…Reversible and efficient genome editing can be enabled by combining CRISPR -Cas system and reverse system for comprehensive gene analysis [24,46]. Simplification of knock in, knock out, transgene and reversible genome engineering which has been achieved in stem cells is done by CRISPR -Cas system [25,24,26,27,46]. There is a acceleration seen in the generation of stem cell lines specially in disease models because of genome editing with CRISPR -Cas system.…”

“…The cause of neurogenerative diseases are neuronal impairments, stem cells models shows appropriate insights in study and cure of neurogenerative disease [28][29][30]46]. Recent study shows that modelling of clinical pathogenic mutations can summarize the specificity of neurogenerative disease for example amyloid β (Aβ)'s generation in Alzheimer's disease (AD) [31][32][33]46]. Validity and safety before clinical applications are lightened up by direct gene correction with CRISPR -Cas system [34,35,46].…”

“…Recent study shows that modelling of clinical pathogenic mutations can summarize the specificity of neurogenerative disease for example amyloid β (Aβ)'s generation in Alzheimer's disease (AD) [31][32][33]46]. Validity and safety before clinical applications are lightened up by direct gene correction with CRISPR -Cas system [34,35,46]. Researchers have used Cas9 to inactive a endogenous retrovirus in pigs [36,47], and also to engineer T cells as a start to develop advanced immunotherapies to target cancers [37,47].…”