Potential of Epigenetic Therapy for Prader-Willi Syndrome

Publication date: Available online 25 July 2019Source: Trends in Pharmacological SciencesAuthor(s): Sung Eun Wang, Yong-hui JiangPrader-Willi syndrome (PWS) is a neurobehavioral and epigenetic disorder caused by the deficiency of paternally expressed genes in the chromosome 15q11-q13. This unique molecular defect renders PWS an exciting opportunity to explore epigenetic therapy. Here, we briefly highlight recent findings from small molecule screening and CRISPR/Cas9-mediated epigenome editing that offer promising therapeutic options along with the challenges that remain in developing a successful epigenetic therapy for PWS in humans.
Source: Trends in Pharmacological Sciences - Category: Drugs & Pharmacology Source Type: research