Potential of Epigenetic Therapy for Prader-Willi Syndrome

Sung Eun Wang; Yong-Hui Jiang

doi:10.1016/j.tips.2019.07.002

Potential of Epigenetic Therapy for Prader-Willi Syndrome

Trends Pharmacol Sci. 2019 Sep;40(9):605-608. doi: 10.1016/j.tips.2019.07.002. Epub 2019 Jul 25.

Authors

Sung Eun Wang¹, Yong-Hui Jiang²

Affiliations

¹ Department of Pediatrics, Duke University of School of Medicine, Durham, NC 27710, USA.
² Department of Pediatrics, Duke University of School of Medicine, Durham, NC 27710, USA; Department of Neurobiology, Duke University of School of Medicine, Durham, NC 27710, USA; Program in Genetics and Genomics, Duke University of School of Medicine, Durham, NC 27710, USA. Electronic address: [email protected].

PMID: 31353046
DOI: 10.1016/j.tips.2019.07.002

Abstract

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.

Keywords: CRISPR/Cas9 mediated epigenome editing; G9a/EHMT2 inhibitor; Prader-Willi syndrome (PWS); epigenetic therapy; genomic imprinting; small molecules.

Publication types

Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

MeSH terms

Epigenesis, Genetic
Epigenomics / methods
Humans
Prader-Willi Syndrome / drug therapy
Prader-Willi Syndrome / genetics*
Prader-Willi Syndrome / therapy*