Muscle-directed gene therapy with adeno-associated viral (AAV) vectors is undergoing
clinical development for treating neuromuscular disorders and for systemic delivery of …

Introduction: Muscle-directed gene therapy is rapidly gaining attention primarily because
muscle is an easily accessible target tissue and is also associated with various severe …

The first in vivo adeno-associated viral vector (AAV) gene transfer experiments were
performed in murine models of muscle directed gene transfer. These studies were …

Adeno-associated virus (AAV) is the most promising gene delivery vehicle for muscle-
directed gene therapy. AAV's natural tropism to muscle cells, long-term persistent transgene …

Adeno-associated viral (AAV) vector-mediated gene replacement for the treatment of
muscular dystrophy represents a promising therapeutic strategy in modern medicine. One …

In the absence of an immune response from the host, intramuscular (IM) injection of
recombinant adeno-associated virus (rAAV) results in the permanent expression of the …

Adeno-associated virus (AAV) is emerging as a vector of choice for muscle gene therapy
because of its effective and stable transduction in striated muscles. AAV naturally evolve into …

Original reports of adeno-associated virus (AAV) vector-mediated gene transfer to the
muscle resulted in high-level β-galactosidase (β-gal) expression and the promise of a viral …

Gene therapy targeting the muscle using adeno-associated vectors (AAV) has a long track
record starting from the first vector design in the 1980s until today where systemic delivery to …

Gene transfer to muscle holds overt promise for the treatment of inherited myopathies,
lysosomal storage disorders, and serum protein deficiencies. In addition, muscle could …