GeneticsbioRxivPreprint
SMCHD1 is a novel target for gene-activation therapy to treat Prader-Willi Syndrome
Megan Iminitoff, Anna K. Le Fevre, Tamara Cameron…·Marnie E. Blewitt
This study identifies SMCHD1 as a target for gene-activation therapy in Prader-Willi Syndrome by showing its role in repressing the PWS gene cluster and demonstrating gene activation in mouse models.
DOI 10.64898/2026.05.13.725051