Potential of Epigenetic Therapy for Prader-Willi Syndrome


Wang SE, Jiang YH

Scientific Notation:

Trends in Pharmacological Sciences

Publication Link:



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.

FPWR Grant:

Preclinical studies of a novel epigenetic therapy for Prader-Willi syndrome