Published online Nov 26, 2019. doi: 10.4252/wjsc.v11.i11.937
Peer-review started: March 23, 2019
First decision: August 1, 2019
Revised: August 12, 2019
Accepted: September 11, 2019
Article in press: September 11, 2019
Published online: November 26, 2019
The identification of new and even more precise technologies for modifying and manipulating the genome has been a challenge since the discovery of the DNA double helix. The ability to modify selectively specific genes provides a powerful tool for characterizing gene functions, performing gene therapy, correcting specific genetic mutations, eradicating diseases, engineering cells and organisms to achieve new and different functions and obtaining transgenic animals as models for studying specific diseases. Clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 technology has recently revolutionized genome engineering. The application of this new technology to stem cell research allows disease models to be developed to explore new therapeutic tools. The possibility of translating new systems of molecular knowledge to clinical research is particularly appealing for addressing degenerative diseases. In this review, we describe several applications of CRISPR/Cas9 to stem cells related to degenerative diseases. In addition, we address the challenges and future perspectives regarding the use of CRISPR/Cas9 as an important technology in the medical sciences.
Core tip: The possibility of translating new molecular knowledge systems to clinical research is particularly appealing for counteracting degenerative diseases as well as infective pathologies and cancer. A novel gene-editing technique, CRISPR/Cas9, has recently emerged for inducing targeted genetic modifications. Therefore, in this review, we describe recent applications of CRISPR/Cas9 to stem cells for counteracting degenerative diseases.