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

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.

Genome Engineering in Livestock: Recent Advances and Regulatory Framework

“…The CRISPR/Cas9-mediated genome engineering technologies allow performing precise genetic modifications, which dramatically enhance the ease and speed for producing genetically modified livestock (Zhao et al, 2019;Van Eenennaam, 2019;Menchaca et al, 2020;Lee et al, 2020;Navarro-Serna et al, 2020;Bishop and Van Eenennaam, 2020;Dua et al, 2021). CRISPR/Cas9 even allowed genome editing in non-human primates, which are hard to tackle (Niu et al, 2014;Kues et al, 2022). In the same year, genome-edited pigs were produced using the CRISPR/Cas9 system in which the von Willebrand factor (vWF) gene was targeted in order to generate a medical model with reduced activity of coagulation factor VIII leading to severe bleeding closely mimicking the human disease (Hai et al, 2014).…”