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pro vyhledávání: '"Saskia Houwen-Opstal"'
Autor:
Annemieke Aartsma-Rus, Liesbeth De Waele, Saskia Houwen-Opstal, Janbernd Kirschner, Yvonne D. Krom, Eugenio Mercuri, Erik H. Niks, Volker Straub, Hermine A. van Duyvenvoorde, Elizabeth Vroom
Publikováno v:
Journal of Neuromuscular Diseases. 10:315-325
Antisense oligonucleotide (ASO) mediated exon skipping aims to reframe dystrophin transcripts for patients with Duchenne muscular dystrophy (DMD). Currently 4 ASOs have been approved by the Food and Drug Administration targeting exon 45, 51 and 53 ba