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Applications of genome editing tools in stem cells towards regenerative medicine: An update

Author(s):

Wilfried A Kues*, Dharmendra Kumar , Naresh L Selokar and Thirumala Rao Talluri*  

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 manipulating of any gene 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, make alterations and cure diseases permanently that are not responding and resistant to traditional therapies. These genome engineering tools have evolved in the field of biomedical research and have also shown to have a significant improvement in clinical trials. However, their widespread use in research revealed potential safety issues, which need to be addressed before implementing such techniques in clinical purposes. Significant and valiant attempts are being 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

Affiliation:

Friedrich-Loeffler-Institut, Federal Research Institute for Animal Health, Department of Biotechnology, Stem Cell Physiology, Höltystr 10, 31535 Neustadt, Animal Physiology and Reproduction Division, ICAR-Central Institute for Research on Buffaloes, Hisar-125001, Haryana, Animal Physiology and Reproduction Division, ICAR-Central Institute for Research on Buffaloes, Hisar-125001, Haryana, Equine Production Campus, ICAR- National Research Centre on Equines, Bikaner-334001, Rajasthan



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