While disruption of alternative splicing underlies many diseases, modulation of splicing
using antisense oligonucleotides (AONs) can have therapeutic implications. The most …

Antisense-mediated modulation of splicing is one of the few fields where antisense
oligonucleotides (AONs) have been able to live up to their expectations. In this approach …

The manipulation of pre-mRNA to alter gene transcript splicing patterns offers considerable
potential for many genetic disorders. In particular, the targeted removal of one or more exons …

Manipulation of pre-mRNA splicing by antisense oligonucleotides (AOs) offers considerable
potential for a number of genetic disorders. One of these is Duchenne muscular dystrophy …

Antisense-mediated exon skipping is an approach that uses antisense oligonucleotides
(AONs) to modulate splicing by hiding specific sites essential for exon inclusion from the …

Antisense-mediated exon skipping is currently the most promising therapeutic approach for
Duchenne muscular dystrophy (DMD). The rationale is to use antisense oligonucleotides …

During the past 10 years, antisense oligonucleotide-mediated exon skipping and splice
modulation have proven to be powerful tools for correction of mRNA splicing in genetic …

Antisense-mediated exon skipping for Duchenne muscular dystrophy (DMD) is currently
tested in phase 3 clinical trials. The aim of this approach is to modulate splicing by skipping …

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) …

In 1995, we were the first to propose antisense oligonucleotide (ASO)-mediated exon-
skipping therapy for the treatment of Duchenne muscular dystrophy (DMD), a noncurable …