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 …

Transposable elements (TEs) or “jumping genes” historically have been disparaged as a
class of “junk DNA” in mammalian genomes (1, 2). The advent of whole genome DNA …

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 …

The ability to efficiently modify the genome using CRISPR technology has rapidly
revolutionized biology and genetics and will soon transform medicine. Duchenne muscular …

Duchenne muscular dystrophy (DMD) is an X-linked disease caused by mutations in the
DMD gene and loss of the protein dystrophin. The absence of dystrophin leads to myofiber …

Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder in which the loss of
dystrophin causes progressive degeneration of skeletal and cardiac muscle. Potential …

Micro-dystrophins are highly promising candidates for treating Duchenne muscular
dystrophy, a lethal muscle disease caused by dystrophin deficiency. Here, we report robust …

Sarcolipin (SLN) is an inhibitor of the sarco/endoplasmic reticulum (SR) Ca2+ ATPase
(SERCA) and is abnormally elevated in the muscle of Duchenne muscular dystrophy (DMD) …

Dystrophin deficiency results in lethal Duchenne muscular dystrophy (DMD). Substituting
missing dystrophin with abbreviated microdystrophin has dramatically alleviated disease in …