Neurodevelopmental disorders: CRISPR to the rescue

Nature Reviews Drug Discovery 17, 240 (2018). doi:10.1038/nrd.2018.39 Author: M. Teresa Villanueva Fragile X syndrome (FXS) is a neurodevelopmental disorder caused by a CGG repeat expansion in the promoter region of FMR1. This mutation results in hypermethylation and silencing of FMR1 and absence, or reduction, of its gene product, fragile X mental retardation protein 1
Source: Nature Reviews Drug Discovery - Category: Drugs & Pharmacology Authors: Tags: Research Highlight Source Type: research