Key result
A cocktail of phosphorodiamidate morpholino oligomers effectively induced dose-dependent skipping of dystrophin exons 45-55 and rescued dystrophin protein in myotubes transdifferentiated from DMD patient fibroblasts.
Why the study?
Does a cocktail of PMOs induce dystrophin exons 45-55 skipping and protein rescue in myotubes transdifferentiated from DMD patient fibroblasts?
Population
Human Duchenne muscular dystrophy patient fibroblast cells harboring out-of-frame deletion mutations of…
Design
Preclinical
Authors
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Hypothesis-generating for multi-exon skipping in DMD; requires in vivo validation before clinical consideration.
Does a cocktail of PMOs induce dystrophin exons 45-55 skipping and protein rescue in myotubes transdifferentiated from DMD patient fibroblasts?
A cocktail of PMOs successfully induced dystrophin exons 45-55 skipping and protein rescue in transdifferentiated DMD patient myotubes, validating this in vitro model and supporting the feasibility of multi-exon skipping therapy.
Lee et al. (2018) studied Duchenne muscular dystrophy (DMD) (n=3). Phosphorodiamidate morpholino oligomer (PMO) cocktails was evaluated on Dose-dependent production of exons 45-55 skipped transcripts and rescue of dystrophin protein. A cocktail of phosphorodiamidate morpholino oligomers effectively induced dose-dependent skipping of dystrophin exons 45-55 and rescued dystrophin protein in myotubes transdifferentiated from DMD patient fibroblasts.