TY - JOUR
T1 - Induced dystrophin exon skipping in human muscle explants
AU - Mcclorey, Graham
AU - Fall, Abbie
AU - Moulton, H.M.
AU - Iversen, P.L.
AU - Rasko, J.E.
AU - Ryan, Monique M.
AU - Fletcher, Susan
AU - Wilton, Steve
PY - 2006
Y1 - 2006
N2 - Antisense oligonucleotide (AO) manipulation of pre-mRNA splicing of the dystrophin gene is showing promise in overcoming Duchenne muscular dystrophy (DMD)-causing mutations. To date, this approach has been limited to studies using animal models or cultured human muscle cells, and evidence that AOs can induce exon skipping in human muscle has yet to be shown. In this study, we used different AO analogues to induce exon skipping in muscle explants derived from normal and DMD human tissue. We propose that inducing exon skipping in human muscle explants is closer to in vivo conditions than cells in monolayer cultures, and may minimize the numbers of participants in Phase I clinical studies to demonstrate proof of principle of exon skipping in human muscle. (C) 2006 Elsevier B.V. All rights reserved.
AB - Antisense oligonucleotide (AO) manipulation of pre-mRNA splicing of the dystrophin gene is showing promise in overcoming Duchenne muscular dystrophy (DMD)-causing mutations. To date, this approach has been limited to studies using animal models or cultured human muscle cells, and evidence that AOs can induce exon skipping in human muscle has yet to be shown. In this study, we used different AO analogues to induce exon skipping in muscle explants derived from normal and DMD human tissue. We propose that inducing exon skipping in human muscle explants is closer to in vivo conditions than cells in monolayer cultures, and may minimize the numbers of participants in Phase I clinical studies to demonstrate proof of principle of exon skipping in human muscle. (C) 2006 Elsevier B.V. All rights reserved.
U2 - 10.1016/j.nmd.2006.05.017
DO - 10.1016/j.nmd.2006.05.017
M3 - Article
C2 - 16919955
SN - 0960-8966
VL - 16
SP - 583
EP - 590
JO - Neuromuscular Disorders
JF - Neuromuscular Disorders
IS - 9-10
ER -