AAV1-CAG-RLuc

A fundamental technique in biomedical research is the delivery of genes of interest (transgenes) into cells to alter their behavior. While transgene delivery can be achieved through a variety of different transfection strategies, such as chemical, lipid, or electroporation-based methods, there are many cell types that cannot be efficiently transfected by these means (e.g., primary T cells, cardiomyocytes, and primary hematopoietic stem cells). Viral vectors have become an important resource for overcoming these barriers to gene transfer. There are many different viral vectors that have been used for gene transfer, including retroviruses, lentiviruses, and adenoviruses, but one of the most commonly used viral vectors is the recombinant adeno-associated virus (AAV).

AAV is a small, non-enveloped virus that packages both negatively and positively charged single-stranded DNA. AAV is a member of the Parvoviridae family and requires a helper virus (e.g., adenovirus or herpes virus) for efficient infection. The wild-type genome is 4.7 kb and contains two major open reading frames (ORFs), including the Rep gene and the Cap gene. In addition, a third ORF has recently been discovered to exist. When using AAV as a gene transfer vector, the endogenous gene is removed and replaced by an expression cassette for the gene of interest.