Antisense oligonucleotide therapy rescues disturbed brain rhythms and sleep in juvenile and adult mouse models of Angelman syndrome
Dongwon Lee, Wu Chen, Heet Naresh Kaku, Xinming Zhuo, Eugene S. Chao, Armand Soriano, Allen Kuncheria, Stephanie Flores, Joo Hyun Kim, Armando M Rivera, Frank Rigo, Paymaan Jafar‐Nejad, Arthur L. Beaudet, Matthew S. Caudill, Mingshan Xue
This study shows that reducing Ube3a-ATS with antisense oligonucleotides rescues EEG rhythms and sleep in Angelman syndrome mouse models, suggesting a broader therapeutic window than previously believed.
eLife · Jan 3, 2023Peer-reviewedNeuroscience