Antisense oligonucleotide (ASO)-mediated exon skipping can restore the open reading
frame of dystrophin transcripts for Duchenne muscular dystrophy (DMD) patients. This …

Duchenne muscular dystrophy (DMD) is the most common childhood neuromuscular
disorder. It is caused by mutations in the DMD gene that disrupt the open reading frame …

Duchenne muscular dystrophy (DMD) is an X-linked disorder characterized by progressive
muscle degeneration, caused by the absence of dystrophin. Exon skipping by antisense …

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

Genetic neuromuscular diseases are caused by defective expression of nuclear or
mitochondrial genes. Mutant genes may reduce expression of wild-type proteins, and …

Splice-modulation therapy, whereby molecular manipulation of premessenger RNA splicing
is engineered to yield genetic correction, is a promising novel therapy for genetic diseases …

Duchenne muscular dystrophy (DMD) is an incurable, X‐linked progressive muscle
degenerative disorder that results from the absence of dystrophin protein and leads to …

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 …

Background Antisense‐mediated exon skipping is a putative treatment for Duchenne
muscular dystrophy (DMD). Using antisense oligonucleotides (AONs), the disrupted DMD …

Research and drug development concerning rare diseases are at the cutting edge of
scientific technology. To date, over 7,000 rare diseases have been identified. Despite their …