Adeno-Associated Virus (AAV) belongs to the Parvoviridae family and is a non-enveloped, single-stranded linear DNA virus.
The AAV genome is approximately 4,700 base pairs long and includes two open reading frames (ORFs) at the upstream and downstream ends, as well as two inverted terminal repeats (ITRs). The ORFs encode two proteins, Cap and Rep, which are involved in viral replication and packaging.
AAV2 is a wild-type AAV with a broad host range. Recombinant adeno-associated virus (rAAV), developed based on wild-type AAV, deletes the coding sequences for AAV proteins and introduces therapeutic gene sequences. It is optimal for carrying genomes under 5 kb. Due to its high safety, low immunogenicity, good viral spread, long-term and stable expression of exogenous genes, physical stability, and strong tissue specificity, rAAV is considered one of the most promising vectors for gene therapy research.
Genomeditech’s AAV-related products consist of an expression plasmid (encoding the gene of interest and two ITR sequences), a helper plasmid (carrying Cap and Rep genes), and an adenovirus helper plasmid (providing adenoviral gene products). These three plasmids are co-transfected into host cells to replicate and package AAV. The resulting viral particles undergo ultracentrifugation purification and titer quantification, ensuring they meet various experimental requirements.