CAG-Cre-GFP AAV (Serotype BR1)

Adeno-associated virus (AAV) is a small, non-pathogenic, replication-defective virus that infects several primate species, including humans. AAV consists of a protein capsid and linear single-stranded DNA encoding genes essential for its life cycle. Recombinant AAVs used to deliver therapeutic transgenes using vectors rarely integrate into the host cell genome and function primarily as episomes. However, a recent canine study demonstrated AAV integration events and clonal expansion. This study highlights the need for long-term monitoring for potential genotoxicity. Currently, AAV is the mainstream delivery method for in vivo gene therapy. AAV-mediated gene therapy has been or is being evaluated in more than two hundred clinical trials. Indications range from central nervous system (CNS), ocular, muscle, and metabolic diseases to aging, HIV, and cocaine use. AAV is composed of 60 highly homogeneous proteins and a single strand of genomic DNA. The capsid of AAV is an icosahedron assembled from 20 VP1 proteins, 20 VP1 proteins, and 20 VP3 proteins; these three protein groups share the same N-terminal sequence. The genome of AAV contains two inverted terminal repeat (ITR) sequences, which consist of enhancers, promoters, gene sequences, and polyA sequences. The two ITR sequences are located at the 5' and 3' ends of the genome and help form the correct genome structure by forming concatemers. To date, AAV has been recognized as non-pathogenic, inducing only mild immune responses, and rarely integrating its DNA into the host genome.