Nature Reviews Drug Discovery
Volume 19 Issue 11, November 2020
Review Article | 05 October 2020
Modulating gene regulation to treat genetic disorders
cis-Regulation therapy (CRT) — modifying the activity of gene-regulatory elements — is emerging as a potential approach to treat genetic diseases. Here, Matharu and Ahituv assess emerging CRT technologies and present proof-of-concept studies in cell and animal models. Key factors to be considered for the translation of CRT into the clinic are discussed.
Navneet Matharu & Nadav Ahituv