Key result
Novel morpholino antisense oligonucleotides targeting DMD exon 51 boost dystrophin expression ~7-fold versus eteplirsen.
Population
Immortalized DMD muscle cells in vitro and mice carrying the human DMD gene in vivo
Comparison
Newly designed morpholino AOs targeting DMD exon 51 vs eteplirsen sequence
Design
Preclinical in vitro and in vivo study
Authors
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Hypothesis-generating for next-generation exon 51 skipping in DMD; prospective human trials needed before clinical adoption.
Effect estimate: up to >12-fold and 7-fold increase
Newly designed morpholino antisense oligonucleotides demonstrated significantly higher efficacy for exon 51 skipping and dystrophin protein rescue compared to eteplirsen in preclinical models.
Echigoya et al. (2017) studied Duchenne muscular dystrophy (DMD). Newly designed morpholino antisense oligonucleotides (AOs) targeting DMD exon 51 vs. Eteplirsen sequence was evaluated on Efficacy of exon 51 skipping and rescue of dystrophin protein expression (up to >12-fold and 7-fold increase). Newly designed morpholino antisense oligonucleotides targeting DMD exon 51 increased exon 51 skipping efficacy and dystrophin protein expression by up to >12-fold and 7-fold compared with eteplirsen.
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