Antisense‐mediated exon skipping aiming for reading frame restoration is currently a
promising therapeutic application for Duchenne muscular dystrophy (DMD). This approach …

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 …

Protein-truncating mutations in the dystrophin gene lead to the most common childhood form
of muscle wasting, Duchenne muscular dystrophy. Becker muscular dystrophy, a condition …

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

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 …

Cell penetrating peptides (CPPs) have been shown to enhance the cellular uptake of
antisense oligonucleotides (AOs). However, the effectiveness of the CPPs for cytoplasmic or …

Objective: Conjugation of arginine-rich cell-penetrating peptide (CPP) to
phosphorodiamidate morpholino oligomers (PMO) has been shown to enhance cytosolic …

Duchenne muscular dystrophy (DMD) afflicts 1 in 5000 newborn males, leading to
progressive muscle weakening and the loss of ambulation between the ages of 8 and 12 …

Charge neutral steric block oligonucleotide analogues, such as peptide nucleic acids (PNA)
or phosphorodiamidate morpholino oligomers (PMO), have promising biological and …

Duchenne and Becker muscular dystrophies are allelic disorders arising from mutations in
the dystrophin gene. Duchenne muscular dystrophy is characterized by an absence of …