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 …

Duchenne muscular dystrophy (DMD) is one of the most common lethal genetic diseases
worldwide, caused by mutations in the dystrophin (DMD) gene. Exon skipping employs short …

Duchenne muscular dystrophy (DMD) is a lethal genetic disorder that most commonly
results from mutations disrupting the reading frame of the dystrophin (DMD) gene. Among …

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 …

Duchenne muscular dystrophy (DMD), the most common lethal genetic disorder, is caused
by mutations in the dystrophin (DMD) gene. Exon skipping is a therapeutic approach that …

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 …

Duchenne muscular dystrophy (DMD) is one of the most common and lethal genetic
disorders, with 20,000 children per year born with DMD globally. DMD is caused by …

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 …

Duchenne muscular dystrophy (DMD) is one of the most common lethal muscle-wasting
disorders affecting young boys caused by mutations in the DMD gene. Exon skipping has …

Introduction It is established that the exon-skipping approach can restore dystrophin in
Duchenne muscular dystrophy (DMD) patients. However, dystrophin restoration levels are …