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 …

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

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

Antisense-mediated splicing modulation is a tool that can be exploited in several ways to
provide a potential therapy for rare genetic diseases. This approach is currently being tested …

Synthetic splice-switching oligonucleotides (SSOs) target nuclear pre-mRNA molecules to
change exon splicing and generate an alternative protein isoform. Clinical trials with two …

The use of splice‐switching antisense therapy is highly promising, with a wealth of pre‐
clinical data and numerous clinical trials ongoing. Nevertheless, its potential to treat a variety …

The process of pre-mRNA splicing is a common and fundamental step in the expression of
most human genes. Alternative splicing, whereby different splice motifs and sites are …

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 …

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 …

The development of effective therapies for neuromuscular disorders such as Duchenne
muscular dystrophy (DMD) is hampered by considerable challenges: skeletal muscle is the …