Mutations in DMD disrupt the reading frame, prevent dystrophin translation, and cause
Duchenne muscular dystrophy (DMD). Here we describe a CRISPR/Cas9 platform …

Dilated cardiomyopathy is the second most common cause for heart failure with no cure
except a high-risk heart transplantation. Approximately 30% of patients harbor heritable …

Genome editing therapy for Duchenne muscular dystrophy (DMD) holds great promise,
however, one major obstacle is delivery of the CRISPR-Cas9/sgRNA system to skeletal …

Background and purpose: Duchenne muscular dystrophy (DMD), a lethal X-linked recessive
muscle dystrophy, is resulted in by different mutations including mostly frame-shifting gross …

Discovery of the CRISPR-Cas (clustered regularly interspaced short palindromic repeat,
CRISPR-associated) system a decade ago has opened new possibilities in the field of …

Although restoration of dystrophin expression via exon skipping in both cardiac and skeletal
muscle has been successfully demonstrated in the mdx mouse, restoration of cardiac …

Delivery of therapeutic transgenes with adeno-associated viral (AAV) vectors for treatment of
myopathies has yielded encouraging results in animal models and early clinical studies …

Muscular dystrophies are debilitating disorders that result in progressive weakness and
degeneration of skeletal muscle. Although the genetic mutations and clinical abnormalities …

Duchenne muscular dystrophy (DMD), a rare genetic disorder characterized by progressive
muscle weakness, is caused by the absence or a decreased amount of the muscle …

Duchenne muscular dystrophy (DMD) is a currently incurable X-linked neuromuscular
disorder, characterized by progressive muscle wasting and premature death, typically as a …