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Article

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

6 Feb 2020

Abstract excerpt

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 mouse models that harbor expanded...

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PMID: 32029588
DOI: 10.1073/pnas.1919197117

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