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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

2023Molecular Therapy38 citationsDOIOpen Access PDF

Topics & Concepts

UBE3AAngelman syndromeBiologyNeuroscienceGeneticsGeneUbiquitinUbiquitin ligaseGenetic Syndromes and ImprintingRNA modifications and cancerPrenatal Screening and Diagnostics
A high-fidelity RNA-targeting Cas13 restores paternal Ube3a expression and improves motor functions in Angelman syndrome mice | Litcius