Research Publications Archive - Foundation for Prader-Willi Research

Potential of Epigenetic Therapy for Prader-Willi Syndrome

Written by Caroline Vrana-Diaz | Sep 26, 2019 5:52:29 PM

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.