AAV2-CMV-hAsCpf1

After the establishment of the first AAV serotype 2 (AAV2) infectious clone in 1982, AAV2 vectors quickly became popular for gene therapy applications due to their lack of pathogenicity, broad infectious range, and ability to establish long-term transgene expression. Recombinant AAV2 vectors have been tested in preclinical studies for a variety of diseases, such as hemophilia, α1 antitrypsin deficiency, cystic fibrosis, Duchenne muscular dystrophy, and rheumatoid arthritis. To date, multiple AAV serotypes and more than 100 AAV variants have been isolated from adenovirus libraries or human/non-human primate tissues. Utilizing alternative AAV serotypes not only allows for lower vector loads (as they may have higher transduction efficiency), but also helps to avoid pre-existing neutralizing antibodies generated by humoral immune responses resulting from natural infection or previous treatment with AAV vectors. In addition, AAV serotypes and variants can serve as templates for designing tissue-targeted capsid structures, thereby expanding and complementing the current range of AAV vectors.