Lack of RAN-mediated toxicity in Huntington’s disease knock-in mice [Neuroscience]

Identification of repeat-associated non-AUG (RAN) translation in trinucleotide (CAG) repeat diseases has led to the emerging concept that CAG repeat diseases are caused by nonpolyglutamine products. Nonetheless, the in vivo contribution of RAN translation to the pathogenesis of CAG repeat diseases remains elusive. Via CRISPR/Cas9-mediated genome editing, we established knock-in...

Source: Proceedings of the National Academy of Sciences - February 24, 2020 Category: Science Authors: Su Yang, Huiming Yang, Luoxiu Huang, Luxiao Chen, Zhaohui Qin, Shihua Li, Xiao-Jiang Li Tags: Biological Sciences Source Type: research