CRISPR/Cas9 gene-editing: Research technologies, clinical applications and ethical considerations

Abstract

Gene therapy carries the potential to treat more than 10,000 human monogenic diseases and benefit an even greater number of complex polygenic conditions. The repurposing of CRISPR/Cas9, an ancient bacterial immune defense system, into a gene-editing technology has armed researchers with a revolutionary tool for gene therapy. However, as the breadth of research and clinical applications of this technology continues to expand, outstanding technical challenges and ethical considerations will need to be addressed before clinical applications become commonplace. Here, we review CRISPR/Cas9 technology and discuss its benefits and limitations in research and the clinical context, as well as ethical considerations surrounding the use of CRISPR gene editing.

Journal

Title: Seminars in Perinatology

Abbreviated Title: Seminars in Perinatology

Publisher: Elsevier BV

ISSN (Print): 01460005

Publication date Created: December 2018

Publication date (Print): December 2018

Volume: 42

Issue: 8

Pages: 487-500

Article

DOI: 10.1053/j.semperi.2018.09.003

PubMed ID: 30482590

Copyright statement: © 2018

License:

https://www.elsevier.com/tdm/userlicense/1.0/

Product

Self URI (article page): https://linkinghub.elsevier.com/retrieve/pii/S0146000518300818