Title: | A phase I/IIa Clinical Trial in Duchenne Muscular Dystrophy using systemically delivered morpholino antisense oligomer to skip exon 53 (SKIP-NMD) |
Journal : | Human gene therapy. Clinical development, 26, 2 |
Authors: | Muntoni F, Author ; Voit T ; Servais L ; Baudin PY ; Straub V ; Mercuri E ; Bain J ; Dickson G ; Vissière D ; Dorveaux E ; Kaye EM ; Duda P ; van't Hoff W |
Material Type: | Article |
Publication Date: | 2015 |
Size: | p 92 |
Languages: | English |
Keywords : | Duchenne muscular dystrophy ; exon skipping ; morpholinos ; phase 1/2 clinical trials |
Pubmed / DOI : | Pubmed : 26086759 / DOI : 10.1089/humc.2015.2528 |