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 …

Promoting the formation of new collateral vessels in ischemic tissues using angiogenic
growth factors (therapeutic angiogenesis) is a an exciting frontier of cardiovascular …

The structure of the adeno-associated virus (AAV-2) has been determined to 3-Å resolution
by x-ray crystallography. AAV is being developed as a vector for gene therapy to treat …

The dystrophin glycoprotein complex (DGC) is a specialization of cardiac and skeletal
muscle membrane. This large multicomponent complex has both mechanical stabilizing and …

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 …

Recombinant adeno-associated virus (rAAV) vectors stably transduce hepatocytes in
experimental animals. Although the vector genomes are found both as extrachromosomes …

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 …

Duchenne muscular dystrophy (DMD) is the most common and lethal genetic muscle
disorder, caused by recessive mutations in the dystrophin gene. One of every 3,500 males …

Systemic gene delivery into muscle has been a major challenge for muscular dystrophy
gene therapy, with capillary blood vessels posing the principle barrier and limiting vector …

Recombinant adeno-associated viruses (AAVs) have been used widely for in vivo gene
therapy. However, adaptive immune responses to AAV have posed a significant hurdle in …