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 …

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 …

Current gene therapy for Duchenne muscular dystrophy (DMD) utilizes adeno-associated
virus (AAV) to deliver micro-dystrophin (µDys), which does not provide full protection for …

Duchenne muscular dystrophy (DMD) is an X-linked recessive, infancy-onset
neuromuscular disorder characterized by progressive muscle weakness and atrophy …

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) …

The use of vectors based on the small parvovirus adeno-associated virus has gained
significant momentum during the past decade. Their high efficiency of transduction of …

The cardiac stroma contains multipotent mesenchymal progenitors. However, lineage
relationships within cardiac stromal cells are poorly defined. Here, we identified heart …

Highlights•Only intravascular delivery can truly change the course of systemic
diseases.•AAV has the capacity to escape from the blood and lead to bodywide gene …

The ultimate goal of muscular dystrophy gene therapy is to treat all muscles in the body.
Global gene delivery was demonstrated in dystrophic mice more than a decade ago using …