Title:Induced Pluripotent Stem Cells in the Era of Precise Genome Editing
Volume: 19
Issue: 3
Author(s): Meeti Punetha, Sheetal Saini, Suman Chaudhary, Prem Singh Yadav, Kristin Whitworth, Jonathan Green, Dharmendra Kumar*Wilfried A. Kues*
Affiliation:
- Animal Physiology and Reproduction Division, ICAR-Central Institute for Research on Buffaloes, Hisar, 125001,
Haryana, India
- Department of
Biotechnology, Friedrich-Loeffler-Institut, Federal Research Institute for Animal Health, Höltystr 10, 31535, Neustadt,
Germany
Keywords:
CRISPR/Cas9, pluripotent stem cells, gene therapy, non-homologous end-joining, DNA, homologous dependent repair.
Abstract: Genome editing has enhanced our ability to understand the role of genetics in a number of
diseases by facilitating the development of more precise cellular and animal models to study pathophysiological
processes. These advances have shown extraordinary promise in a multitude of areas, from basic
research to applied bioengineering and biomedical research. Induced pluripotent stem cells (iPSCs) are
known for their high replicative capacity and are excellent targets for genetic manipulation as they can be
clonally expanded from a single cell without compromising their pluripotency. Clustered, regularly interspaced
short palindromic repeats (CRISPR) and CRISPR/Cas RNA-guided nucleases have rapidly become
the method of choice for gene editing due to their high specificity, simplicity, low cost, and versatility.
Coupling the cellular versatility of iPSCs differentiation with CRISPR/Cas9-mediated genome editing
technology can be an effective experimental technique for providing new insights into the therapeutic use
of this technology. However, before using these techniques for gene therapy, their therapeutic safety and
efficacy following models need to be assessed. In this review, we cover the remarkable progress that has
been made in the use of genome editing tools in iPSCs, their applications in disease research and gene
therapy as well as the hurdles that remain in the actual implementation of CRISPR/Cas systems.