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Potential of Epigenetic Therapy for Prader-Willi Syndrome
Sung Eun Wang1, Yong-Hui Jiang2
1Department of Pediatrics, Duke University of School of Medicine, Durham, NC 27710, USA.
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.
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