AAV1-EF1α-GFP

Adeno-associated virus (AAV) is a small naked icosahedral virus first discovered in 1965. In addition to being a virus containing single-stranded DNA, AAV remains the only virus that has not been confirmed as a pathogen of any human disease to date. Instead, recombinant AAV vectors have been used in many Phase I/II clinical trials and, in some cases, have shown clinical efficacy in potential gene therapy for a variety of human diseases. In recent years, many other AAV serotypes have been isolated, and their use as vectors may further greatly expand their optimal use for therapeutic purposes.

Several AAV serotypes have been isolated from tissue culture, humans, and non-human primates. Following their development into recombinant vectors, their efficacy has been evaluated in various tissue culture cell lines. To date, 13 different AAV serotype vectors (AAV1–AAV13) have been described, but this number is certain to increase. AAV vector DNA rarely integrates into the host cell genome but can lead to sustained, long-term transgene expression in non-dividing cells. This makes AAV particularly suitable for gene therapy in primarily postmitotic tissues such as the brain, retina, liver, skeletal muscle, or heart.