An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases.

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作者： Merryl,Rodrigues ^[1] ; Toshifumi,Yokota ^[2]

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

关键词 CRISPR Cas9/Cpf1 genome editing Duchenne/Becker muscular dystrophy (DMD/BMD)Dystrophic epidermolysis bullosa (DEB)Eteplirsen (Exondys 51)Exon skipping Fibrodysplasia ossificans progressiva (FOP)Laminin-alpha 2 Myotonic dystrophy Nusinersen (Spinraza)Usher syndrome

主题词动物(Animals);外显子(Exons);基因打靶(Gene Targeting);遗传性疾病, 先天性(Genetic Diseases, Inborn);人类(Humans);肌营养不良(Muscular Dystrophies);RNA剪接(RNA Splicing)

DOI 10.1007/978-1-4939-8651-4_2

PMID 30171533

发布时间 2019-04-01

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