medicalxpress.com· Medical Xpress - Genetics·· 22 hours agoPickScore66
Switching on healthy silent genes reveals new way to treat Prader-Willi syndrome
In brief
A WEHI team reports in Science Advances that removing SMCHD1 can reactivate genes silenced in Prader-Willi syndrome, with gene activity restored in patient-derived stem cells and in animal models.
Why it matters
The study used CRISPR to remove SMCHD1 and combined stem-cell and genomic technologies to track thousands of genes, providing a method to assess whether reactivating dormant genes disrupts other tissues.
Source: medicalxpress.com · medicalxpress.com