Induced dystrophin exon skipping in human muscle explants
G Mcclorey, AM Fall, HM Moulton, PL Iversen… - Neuromuscular …, 2006 - Elsevier
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 …
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 …
