A high-fidelity RNA-targeting Cas13 restores paternal Ube3a expression and improves motor functions in Angelman syndrome mice
Jinhui Li, Zhixin Shen, Yajing Liu, Zixiang Yan, Yuanhua Liu, Xiang Lin, Junjie Tang, Ruimin Lv, Guannan Geng, Zhi‐Qi Xiong, Changyang Zhou, Hui Yang
Topics & Concepts
UBE3AAngelman syndromeBiologyNeuroscienceGeneticsGeneUbiquitinUbiquitin ligaseGenetic Syndromes and ImprintingRNA modifications and cancerPrenatal Screening and Diagnostics