Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study.

BACKGROUND: Mutations that disrupt the open reading frame and prevent full translation of DMD, the gene that encodes dystrophin, underlie the fatal X-linked disease Duchenne muscular dystrophy. Oligonucleotides targeted to splicing elements (splice switching oligonucleotides) in DMD pre-mRNA can lea...

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Bibliographic Details
Main Authors:	Kinali, M, Arechavala-Gomeza, V, Feng, L, Cirak, S, Hunt, D, Adkin, C, Guglieri, M, Ashton, E, Abbs, S, Nihoyannopoulos, P, Garralda, M, Rutherford, M, McCulley, C, Popplewell, L, Graham, I, Dickson, G, Wood, M, Wells, D, Wilton, S, Kole, R, Straub, V, Bushby, K, Sewry, C, Morgan, J, Muntoni, F
Format:	Journal article
Language:	English
Published:	2009

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study.

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