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DUX4 Transcript Knockdown with Antisense 2′-O-Methoxyethyl Gapmers for the Treatment of Facioscapulohumeral Muscular Dystrophy

Kenji Rowel Q. Lim, Adam J. Bittel, Rika Maruyama, Yusuke Echigoya, Quynh Nguyen, Yiqing Huang, Kasia Dzierlega, Aiping Zhang, Yiwen Chen, Toshifumi Yokota

2020Molecular Therapy34 citationsDOIOpen Access PDF

Topics & Concepts

Facioscapulohumeral muscular dystrophyGene knockdownMuscular dystrophyBiologyMessenger RNAMolecular biologyIn vivoSkeletal muscleCell biologyIn vitroGeneGeneticsEndocrinologyMuscle Physiology and DisordersRNA Research and SplicingNeurogenetic and Muscular Disorders Research
DUX4 Transcript Knockdown with Antisense 2′-O-Methoxyethyl Gapmers for the Treatment of Facioscapulohumeral Muscular Dystrophy | Litcius