WEHI team shows SMCHD1 removal reactivates silent genes in Prader-Willi syndrome
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Researchers at WEHI report in Science Advances that removing the protein SMCHD1 can switch back on genes that are silenced in Prader-Willi syndrome, a genetic disorder. Restored gene activity was observed in patient-derived stem cells and in animal models. The approach points to a potential new treatment strategy distinct from managing symptoms. The findings establish the mechanism in laboratory systems; questions such as how the effect could be translated into a therapy for patients, and whether it is safe and effective in humans, remain open.
Written by a model from the reports · updated 4 hours ago
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- medicalxpress.comPickSwitching on healthy silent genes reveals new way to treat Prader-Willi syndrome
A WEHI team publishing in Science Advances shows that removing SMCHD1 can reactivate genes silenced in Prader-Willi syndrome, with restored gene activity observed both in patient-derived stem cells and in animal models.
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