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Autor:
Annemieke Aartsma-Rus, Begoña Aguilera, Ruurd C. Verheul, Christa L Tanganyika-de Winter, Valeriano Diaz Parillas, Silvana M.G. Jirka, Peter C de Visser, Peter A C 't Hoen
Publikováno v:
Molecular Therapy
Molecular Therapy, 26, 1, pp. 132-147
Molecular Therapy, 26(1), 132-147
Molecular Therapy, 26, 132-147
Molecular Therapy, 26, 1, pp. 132-147
Molecular Therapy, 26(1), 132-147
Molecular Therapy, 26, 132-147
Duchenne muscular dystrophy (DMD) is a severe, progressive muscle wasting disorder caused by reading frame disrupting mutations in the DMD gene. Exon skipping is a therapeutic approach for DMD. It employs antisense oligonucleotides (AONs) to restore