Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by dystrophin gene
mutation. Conceptually, replacing the mutated gene with a normal one would cure the …

Heart disease is a major cause of death worldwide with increasing prevalence, which urges
the development of new therapeutic strategies. Over the last few decades, numerous small …

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 …

Abstract Adeno-associated virus (AAV)-mediated CRISPR-Cas9 editing holds promise to
treat many diseases. The immune response to bacterial-derived Cas9 has been speculated …

Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …

The mammalian heart is responsible for not only pumping blood throughout the body but
also adjusting this pumping activity quickly depending upon sudden changes in the …

Adeno-associated viruses (AAVs) are small, nonenveloped single-stranded DNA viruses
that require helper viruses to facilitate efficient replication. Despite the presence of humoral …

Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by
the absence of dystrophin, a membrane-stabilizing protein encoded by the DMD gene …

Duchenne muscular dystrophy (DMD) is a severe, progressive, and ultimately fatal disease
of skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy. The identification …

Duchenne muscular dystrophy (DMD) has been a major target for gene therapy
development for nearly 30 years. DMD is among the most common genetic diseases, and …