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

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 …

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 …

Exon skipping is a therapeutic approach that is feasible for various genetic diseases and
has been studied and developed for over two decades. This approach uses antisense …

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 …

Introduction: Antisense nucleic acid analogues can interact with pre-mRNA motifs and
influence exon or splice site selection and thereby alter gene expression. Design of …

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 …

Introduction: Antisense oligonucleotide (AON) therapy is a form of treatment for genetic or
infectious diseases using small, synthetic DNA-like molecules called AONs. Recent …

Introduction: Exon skipping is a therapeutic approach for Duchenne muscular dystrophy
(DMD) that has been in development for close to two decades. This approach uses …