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Current Stem Cell Research & Therapy

Author(s): Wilfried A. Kues, Dharmendra Kumar , Naresh L. Selokar and Thirumala Rao Talluri*

DOI: 10.2174/1574888X16666211124095527

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Applications of Genome Editing Tools in Stem Cells Towards Regenerative Medicine: An Update

Page: [267 - 279] Pages: 13

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Abstract

Precise and site-specific genome editing through application of emerging and modern gene engineering techniques, namely zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeats (CRISPR/ Cas9) have swiftly progressed the application and use of the stem cell technology in the sphere of in-vitro disease modelling and regenerative medicine. Genome editing tools facilitate the manipulation of genes in various types of cells with target-specific nucleases. These tools aid in elucidating the genetics and etiology behind different diseases and have immense promise as novel therapeutics for correcting the genetic mutations, making alterations, and curing diseases permanently, which are not responding and resistant to traditional therapies. These genome engineering tools have evolved in the field of biomedical research and have also been shown to have a significant improvement in clinical trials. However, their widespread use in the research revealed potential safety issues, which need to be addressed before implementing such techniques for clinical purposes. Significant and valiant attempts need to be made in order to surpass those hurdles. The current review outlines the advancements of several genome engineering tools and describes suitable strategies for their application towards regenerative medicine.

Keywords: Designer nuclease, pluripotent stem cell, genome editing, CRISPR/Cas9, regenerative medicine, zinc finger nucleases (ZFNs).

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