Transcriptional reprogramming restores UBE3A brain-wide and rescues behavioral phenotypes in an Angelman Syndrome mouse model.

O'Geen H, Beitnere U, Garcia MS, Adhikari A, Cameron DL, Fenton TA, Copping NA, Deng P, Lock S, Halmai J, et al. Transcriptional reprogramming restores UBE3A brain-wide and rescues behavioral phenotypes in an Angelman Syndrome mouse model. Mol Ther. 2023.