Defined d-hexapeptides bind CUG repeats and rescue phenotypes of myotonic dystrophy myotubes in a Drosophila model of the disease

Abstract In Myotonic Dystrophy type 1 (DM1), a non-coding CTG repeats rare expansion disease; toxic double-stranded RNA hairpins sequester the RNA-binding proteins Muscleblind-like 1 and 2 (MBNL1 and 2) and trigger other DM1-related pathogenesis pathway defects. In this paper, we characterize four d...

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主要な著者:	Anna Rapisarda, Ariadna Bargiela, Beatriz Llamusi, Isabel Pont, Roger Estrada-Tejedor, Enrique Garcia-España, Ruben Artero, Manuel Perez-Alonso
フォーマット:	Artigo
言語:	Inglês
出版事項:	Nature Portfolio 2021-09-01
シリーズ:	Scientific Reports
オンライン･アクセス:	https://doi.org/10.1038/s41598-021-98866-0
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