A joint research team led by the Keio University Regenerative Medicine Research Center, Keio University School of Medicine and Tokyo Medical University has succeeded in restoring the function of genes silenced in Prader-Willi syndrome, a congenital imprinting disorder. Using patient-derived induced pluripotent stem cells and a CRISPR/Cas9-based epigenome editing system, the researchers demonstrated that previously inactive maternal genes could be reactivated.
This groundbreaking achievement provides a novel therapeutic strategy at the molecular level for PWS, a disorder with no established curative treatment to date. The findings may also pave the way for new approaches to other imprinting disorders. The study was published in Nature Communications on October 28, 2025.
