Duchenne muscular dystrophy (DMD) is a severe genetic disease caused by the loss of the
dystrophin protein. Exon skipping is a promising strategy to treat DMD by restoring truncated …

With the food and drug administration (FDA) approval of nusinersen (trade name Spinraza)
for treatment of spinal muscular atrophy patients on December 23, 2016 [1] brought us not …

Antisense oligonucleotides (ASOs) can modulate pre-mRNA splicing. This offers therapeutic
opportunities for numerous genetic diseases, often in a mutation-specific and sometimes …

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 …

Restoration of the open reading frame of the DMD gene and dystrophin protein production in
Duchenne muscular dystrophy (DMD) can be achieved by exon skipping using antisense …

Duchenne muscular dystrophy (DMD) is a fatal muscle wasting disorder caused by
mutations in the dystrophin gene. In most cases, the open-reading frame is disrupted which …

Alternative splicing is an important regulator of the transcriptome. However, mutations may
cause alteration of splicing patterns, which in turn leads to disease. During the past 10 …

Abstract Background Antisense oligonucleotides (AOs) can interfere with exon recognition
and intron removal during pre-mRNA processing, and induce excision of a targeted exon …

Exon skipping is one of the more promising therapeutic options for Duchenne Muscular
Dystrophy (DMD). The idea is to use antisense oligonucleotides to splice out selected exons …

Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene that
result in the absence of functional protein. In the majority of cases these are out-of-frame …