Introduction Gene therapy provides the exciting opportunity of a curative single treatment for
devastating diseases, eradicating the need for chronic medication. Adeno-associated …

In most cases, pharmacologic strategies to treat genetic muscle disorders and certain
acquired disorders, such as sporadic inclusion body myositis, have produced modest …

Long-term cures of hemophilia B have been achieved using AAV2 delivering the factor IX
gene to the liver of adeno-associated virus (AAV)–naive hemophilic animals. However, the …

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

See page 1040 Adeno-associated virus–based vector (AAV)–mediated gene delivery has
been successful in some animal models of human disease such as the mdx mouse model of …

Although adeno-associated virus (AAV)-mediated gene therapy has been hindered by the
small viral packaging capacity of the vector, trans-splicing AAV vectors are able to package …

Approximately two‐thirds of Duchenne muscular dystrophy (DMD) patients show intragenic
deletions ranging from one to several exons of the DMD gene and leading to a premature …

Duchenne muscular dystrophy (DMD) is a rare, X-linked, fatal, degenerative neuromuscular
disease caused by mutations in the DMD gene. More than 2,000 mutations of the DMD gene …

Limited packaging capacity hinders adeno-associated virus (AAV) gene therapy. A recent
study seems to have provided a solution to this problem. Allocca et al. reported that AAV-5 …

ADENO-ASSOCIATED VIRUS (AAV) VECTORS were first devel-oped 20 years ago (Carter,
2004) and an AAV vector was first administered to a human subject in November 1995 …