Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein.
Improvements in patient care and disease management have slowed down disease …

Dilated cardiomyopathy (DCM) is an umbrella term entailing a wide variety of genetic and
non-genetic etiologies, leading to left ventricular systolic dysfunction and dilatation, not …

Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and
function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD …

Genome editing with CRISPR/Cas9 is a promising new approach for correcting or mitigating
disease-causing mutations. Duchenne muscular dystrophy (DMD) is associated with lethal …

Frameshift mutations in the DMD gene, encoding dystrophin, cause Duchenne muscular
dystrophy (DMD), leading to terminal muscle and heart failure in patients. Somatic gene …

Recent advances in base editing have created an exciting opportunity to precisely correct
disease-causing mutations. However, the large size of base editors and their inherited off …

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

Since its mechanism discovery in 2012 and the first application for mammalian genome
editing in 2013, CRISPR-Cas9 has revolutionized the genome engineering field and created …

Adeno-associated virus (AAV) has shown promising therapeutic efficacy with a good safety
profile in a wide range of animal models and human clinical trials. With the advent of …

Background Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular
childhood disorder that causes progressive muscle weakness and degeneration and results …