Recent medical advances have exploited the ability to address a given disease at the
underlying level of transcription and translation. These treatment paradigms utilize nucleic …

Muscular dystrophies are a heterogeneous group of inherited disorders that share similar
clinical features and dystrophic changes on muscle biopsy. An improved understanding of …

Gene-based therapeutics hold great promise for medical advancement and have been used
to treat various human diseases with mixed success. However, their therapeutic application …

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 …

Leber congenital amaurosis (LCA) is a severe hereditary retinal dystrophy responsible for
congenital or early-onset blindness. The most common disease-causing mutation (> 10%) is …

The development of effective therapies for neuromuscular disorders such as Duchenne
muscular dystrophy (DMD) is hampered by considerable challenges: skeletal muscle is the …

Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of
dilated cardiomyopathy (DCM), a heart disease characterized by ventricular dilatation …

The emergence and spread of antimicrobial-resistant bacterial, viral, and fungal pathogens
for which diminishing treatment options are available is of major global concern. New viral …

Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder resulting from
lesions of the gene encoding dystrophin. These usually consist of large genomic deletions …

Duchenne muscular dystrophy is a rare, progressive, muscle-wasting disease leading to
severe disability and premature death. Treatment is currently symptomatic, but several …