Casimersen for Duchenne muscular dystrophy.

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作者： H,Wilton-Clark ^[1] ; T,Yokota ^[2]

作者单位： Department of Medicine, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, Alberta, Canada. ^[1] Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, Alberta, Canada. toshifum@ualberta.ca. ^[2] The Friends of Garret Cumming Research and Muscular Dystrophy Canada HM Toupin Neurological Science Research Chair, Edmonton, Alberta, Canada. ^[3]

关键词 Amondys-45 Antisense therapy Casimersen DMD expression inhibitors Duchenne muscular dystrophy Exon skipping therapy Gene therapy Phosphorodiamidate morpholino oligomers

主题词外显子(Exons);人类(Humans);男(雄)性(Male);肌营养不良, 杜氏(Muscular Dystrophy, Duchenne);寡核苷酸类(Oligonucleotides);寡核苷酸类, 反义(Oligonucleotides, Antisense)

DOI 10.1358/dot.2021.57.12.3352740

PMID 34909800

发布时间 2021-12-17

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准确一般字义偏差语意偏离译文作用小提交