The application of recombinant DNA technology to prenatal diagnosis of many recessively
inherited X-linked diseases is complicated by a high frequency of heterogenous, new …

Cells derived from blood vessels of human skeletal muscle can regenerate skeletal muscle,
similarly to embryonic mesoangioblasts. However, adult cells do not express endothelial …

A major obstacle limiting gene therapy for diseases of the heart and skeletal muscles is an
inability to deliver genes systemically to muscles of an adult organism. Systemic gene transfer …

Attempts to develop gene therapy for Duchenne muscular dystrophy (DMD) have been
complicated by the enormous size of the dystrophin gene. We have performed a detailed …

BACKGROUND X-linked cardiomyopathy (XLCM) is a rapidly progressive primary myocardial
disorder presenting in teenage males as congestive heart failure. Manifesting female …

Genetic defects in a number of components of the dystrophin–glycoprotein complex (DGC)
lead to distinct forms of muscular dystrophy. However, little is known about how alterations in …

Gene replacement therapies utilizing adeno-associated viral (AAV) vectors hold great promise
for treating Duchenne muscular dystrophy (DMD). A related approach uses AAV vectors …

Dystrophin, the product of the Duchenne muscular dystrophy gene, is tightly associated with
the sarcolemmal membrane to a large glycoprotein complex. One function of the dystrophin-…

Sarcolemma-associated neuronal NOS (nNOS) plays a critical role in normal muscle
physiology. In Duchenne muscular dystrophy (DMD), the loss of sarcolemmal nNOS leads to …

Duchenne muscular dystrophy (DMD) is the most common, lethal genetic disorder of children.
A number of animal models of muscular dystrophy exist, but the most effective model for …