Duchenne muscular dystrophy (DMD) is a severe muscle‐wasting disease caused by frame
shifting and nonsense mutations in the dystrophin gene. Through skipping of an (additional) …

Duchenne muscular dystrophy (DMD), a fatal X-linked recessive disorder, is caused mostly
by frame-disrupting, out-of-frame deletions in the dystrophin (DMD) gene. Antisense …

Through antisense-induced single-, double-, and multiexon skipping, we have previously
demonstrated restoration of dystrophin expression in Duchenne muscular dystrophy (DMD) …

Antisense oligonucleotide (AO)-mediated splice modulation has been established as a
therapeutic approach for tackling genetic diseases. Recently, Exondys51, a drug that aims to …

As small molecule drugs for Duchenne muscular dystrophy (DMD), antisense
oligonucleotides (AONs) have been shown to restore the disrupted reading frame of DMD …

Twenty-seven participants from 9 countries (Australia; Belgium; England; France; Germany;
Italy; Japan; The Netherlands; USA) attended the third ENMC workshop on exon skipping …

Background The specific skipping of an exon, induced by antisense oligonucleotides (AON)
during splicing, has shown to be a promising therapeutic approach for Duchenne muscular …

Splice-switching oligonucleotides (SSOs) are short, synthetic, antisense, modified nucleic
acids that base-pair with a pre-mRNA and disrupt the normal splicing repertoire of the …

Protein-truncating mutations in the dystrophin gene lead to the most common childhood form
of muscle wasting, Duchenne muscular dystrophy. Becker muscular dystrophy, a condition …

In the past few years, research in targeted mutation therapies has experienced significant
advances, especially in the field of rare diseases. In particular, the efficacy of antisense …