scAAV1-CAG-GFP

AAV virions are small (~20 nm), naturally replication-defective, non-enveloped viruses that are roughly spherical in shape. Wild-type AAV can infect both dividing and non-dividing cells and can remain latent in host cell DNA by integrating into the host cell genome in a site-specific manner (the AAVS1 locus on human chromosome 19). Recombinant AAV (rAAV) is widely used in neuroscience due to its broad tropism, relatively long-term expression in non-dividing cells (including neurons), and lack of pathogenicity in animal models. Unlike wild-type AAV, the genome of rAAV vectors does not typically integrate into host DNA but is instead maintained primarily in a circular form (called episomes) in the nucleus of transduced cells.

Viral titer and capsid-receptor interactions are key factors determining the efficiency with which AAV enters cells and sheds single-stranded DNA. The subsequent synthesis of the complementary strand by the host cell machinery is a slow process that affects rAAV transgene expression and transduction efficiency. Dimeric or self-complementary AAV (scAAV) was designed to overcome this limitation. Compared with single-stranded rAAV, scAAV vectors have 5- to 140-fold higher transduction efficiencies in vitro and enable rapid and higher-level expression of gene payload in vivo.