CRISPR/Cas9 directed to the Ube3a antisense transcript improves Angelman syndrome phenotype in mice

This study provides compelling evidence to further investigate editing of the homologous region of the human UBE3A-ATS because this may provide a lasting therapeutic effect for patients with AS.

Source: Journal of Clinical Investigation - March 2, 2021 Category: Biomedical Science Authors: Ralf S. Schmid, Xuefeng Deng, Priyalakshmi Panikker, Msema Msackyi, Camilo Breton, James M. Wilson Source Type: research