Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …

Duchenne muscular dystrophy (DMD) is a debilitating and fatal genetic disease affecting
1/5000 boys globally, characterized by progressive muscle breakdown and eventual death …

Muscular dystrophies (MDs) comprise a diverse group of inherited disorders characterized
by progressive muscle loss and weakness. Given the genetic etiology underlying MDs …

Myotonic dystrophy type 1 (DM1) is a dominant genetic disease in which the expansion of
long CTG trinucleotides in the 3′ UTR of the myotonic dystrophy protein kinase (DMPK) …

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

Introduction Duchenne muscular dystrophy (DMD) is one of the most severe and devastating
neuromuscular hereditary diseases with a male newborn incidence of 20 000 cases each …

Duchenne muscular dystrophy (DMD) is a devastating disease that results in life-limiting
complications such as loss of skeletal muscle function as well as respiratory and cardiac …

Duchenne muscular dystrophy (DMD) is a progressive X-linked disease caused by
mutations in the DMD gene that prevent the expression of a functional dystrophin protein …

Abstract Adeno‐associated virus (AAV)‐based therapies are recognized as one of the most
potent next‐generation treatments for inherited and genetic diseases. However, several …

Neurological and neuromuscular diseases resulting from familial, sporadic, or de novo
mutations have devasting personal, familial, and societal impacts. As the initial product of …