Induced Pluripotent Stem Cells in the Era of Precise Genome Editing

Author:

Punetha Meeti1ORCID,Saini Sheetal1ORCID,Chaudhary Suman1,Yadav Prem Singh1,Whitworth Kristin2,Green Jonathan2,Kumar Dharmendra1ORCID,Kues Wilfried A.3ORCID

Affiliation:

1. Animal Physiology and Reproduction Division, ICAR-Central Institute for Research on Buffaloes, Hisar, 125001, Haryana, India

2. Division of Animal Sciences, University of Missouri, Columbia, MO, 65211, USA

3. Department of Biotechnology, Friedrich-Loeffler-Institut, Federal Research Institute for Animal Health, Höltystr 10, 31535, Neustadt, Germany

Abstract

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.

Funder

ICAR-National Agricultural Science Fund, New Delhi, India

Publisher

Bentham Science Publishers Ltd.

Subject

General Medicine,Medicine (miscellaneous)

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