CAG repeat-binding small molecule improves motor coordination impairment in a mouse model of Dentatorubral–pallidoluysian atrophy

CAG repeat-binding small molecule improves motor coordination impairment in a mouse model of Dentatorubral–pallidoluysian atrophy

Dentatorubral–pallidoluysian atrophy (DRPLA) is a devastating genetic disease presenting myoclonus, epilepsy, ataxia, and dementia. DRPLA is caused by the expansion of a CAG repeat in the ATN1 gene. Aggregation of the polyglutamine-expanded ATN1 protein causes neuro-degeneration of the dentatorubral...

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Bibliographic Details
Main Authors: Yuhei Hasuike, Hana Tanaka, Terence Gall-Duncan, Mustafa Mehkary, Kazuhiko Nakatani, Christopher E. Pearson, Shoji Tsuji, Hideki Mochizuki, Masayuki Nakamori
Format: Article
Language:English
Published: Elsevier 2022-02-01
Series:Neurobiology of Disease
Subjects:
Dentatorubral–pallidoluysian atrophy
Naphthyridine–azaquinolone
Repeat expansion
Repeat instability
Trinucleotide
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996121003533

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