Induced Pluripotent Stem Cells in the Era of Precise Genome Editing

Meeti      Punetha; Sheetal      Saini; Suman      Chaudhary; Prem   Singh   Yadav; Kristin      Whitworth; Jonathan      Green; Dharmendra      Kumar; Wilfried   A.   Kues
doi:10.2174/1574888X18666230307115326
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.
Keywords: CRISPR/Cas9, pluripotent stem cells, gene therapy, non-homologous end-joining, DNA, homologous dependent repair.
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DOI https://dx.doi.org/10.2174/1574888X18666230307115326	Print ISSN 1574-888X
Publisher Name Bentham Science Publisher	Online ISSN 2212-3946
Current Stem Cell Research & Therapy

Induced Pluripotent Stem Cells in the Era of Precise Genome Editing

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