AAV9-CMV-Luc-PEST

Viral vectors are highly evolved gene delivery systems for therapeutic interventions. In the past decade, adeno-associated viruses (AAV) have emerged as one of the most promising vectors for gene therapy and vaccines. The safety and efficacy of AAV vectors have been evaluated in clinical trials for the treatment of different diseases, including Parkinson's disease, cystic fibrosis, hemophilia B, rheumatoid arthritis, Leber's congenital amaurosis, and lipoprotein lipase deficiency. Recombinant AAV (rAAV) is generated by replacing the entire viral genome, except for the ITRs, with the desired genetic payload. The genetic payload is flanked by the ITRs, which are essential for genome encapsidation and subsequent vector production. Early observations that more than 100,000 rAAV particles could be produced per cell suggested that the vector was non-pathogenic and could be used for gene therapy. This inspired decades of research that put this helper-dependent virus at the center of the gene therapy field. rAAV has several properties that are beneficial for gene therapy. These properties include the ability to transduce both dividing and non-dividing cells and to maintain long-term transgene expression, primarily in slowly dividing cells. AAV serotypes with multiple cell tropisms allow one to target a wide range of diseases. In addition, the field of AAV vectorology has now advanced to the stage where capsids can be tailored to transduce specific cell types.