Title:Applications of Genome Editing Tools in Stem Cells Towards Regenerative Medicine: An Update
Volume: 17
Issue: 3
Author(s): Wilfried A. Kues, Dharmendra Kumar , Naresh L. Selokar and Thirumala Rao Talluri*
Affiliation:
- Department of Animal Reproduction, ICAR-National Research Centre
on Equines, Equine Production Campus, ICAR-NRC on Equines, Bikaner, Rajasthan, India
Keywords:
Designer nuclease, pluripotent stem cell, genome editing, CRISPR/Cas9, regenerative medicine, zinc finger nucleases (ZFNs).
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.