Adeno-associated virus (AAV) is a small, nonenveloped virus that was adapted 30 years
ago for use as a gene transfer vehicle. It is capable of transducing a wide range of species …

A resurgence of interest and investment in the field of gene therapy, driven in large part by
advances in viral vector technology, has recently culminated in United States Food and Drug …

The concept behind gene therapy is simple—by delivering corrective genetic material to the
cells of a patient the symptoms of disease can be alleviated. But seven years after the first …

Conventional methods for rAAV purification that are based on cesium chloride
ultracentrifugation have often produced vector preparations of variable quality and resulted …

Recently, efficient and long-term in vivo gene transfer by recombinant adeno-associated
virus type 2 (rAAV) vectors has been demonstrated in a variety of tissues. Further …

The large number of candidate genes made available by comprehensive genome analysis
requires that relatively rapid techniques for the study of function be developed. Here, we …

The relationship between the neurosensory photoreceptors and the adjacent retinal pigment
epithelium (RPE) controls not only normal retinal function, but also the pathogenesis of …

The death of photoreceptor cells caused by retinal degenerative diseases often results in a
complete loss of retinal responses to light. We explore the feasibility of converting inner …

Recombinant adeno-associated virus 2 (rAAV2) has been shown to deliver genes to
neurons effectively in the brain, retina, and spinal cord. The characterization of new AAV …

Recombinant adeno-associated viruses (rAAV) are produced transiently in mammalian cells
usually by cotransfecting two or three plasmids containing AAV genes, adenovirus helper …