Potential of Epigenetic Therapy for Prader-Willi Syndrome.


Journal Article

Prader-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.

Full Text

Cited Authors

  • Wang, SE; Jiang, Y-H

Published Date

  • September 2019

Published In

Volume / Issue

  • 40 / 9

Start / End Page

  • 605 - 608

PubMed ID

  • 31353046

Pubmed Central ID

  • 31353046

Electronic International Standard Serial Number (EISSN)

  • 1873-3735

International Standard Serial Number (ISSN)

  • 0165-6147

Digital Object Identifier (DOI)

  • 10.1016/j.tips.2019.07.002


  • eng