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 …

Duchenne muscular dystrophy (DMD) is a lethal disorder caused by mutations in the DMD
gene. Antisense-mediated exon-skipping is a promising therapeutic strategy that makes use …

Antisense-mediated exon skipping and exon inclusion have proven to be powerful tools for
treating neuromuscular diseases. The approval of Exondys 51 (eteplirsen) and Spinraza …

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 …

Clinical implementation of two recently approved antisense RNA therapeutics–Exondys51®
to treat Duchenne muscular dystrophy (Duchenne MD) and Spinraza® as a treatment for …

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 …

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

Duchenne muscular dystrophy (DMD) is one of the most common and lethal genetic
disorders, with 20,000 children per year born with DMD globally. DMD is caused by …

Background Targeted splice modulation of pre‐mRNA transcripts by antisense
oligonucleotides (AOs) can correct the function of aberrant disease‐related genes …

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