scAAV DJ/8-Cre

Adeno-associated viruses (AAV) are DNA viruses belonging to the Parvoviridae family. They consist of a small, non-enveloped icosahedral capsid (T1 symmetry) with a diameter of 26 nm. The 4.7 kb linear single-stranded DNA genome contains two structural genes (Rep and Cap) with an inverted terminal repeat (ITR) of 145 nt at each end. Replacement of the rep and cap genes with the transgene of interest converts AAV into recombinant AAV (rAAV) vectors that efficiently transduce the liver, pancreas, heart, lung, striated muscle, and central nervous system, making rAAV-mediated gene delivery a powerful approach for research and clinical applications.

The first generation of rAAV vectors consisted of a single-stranded DNA genome that was transcriptionally inactive until it became double-stranded in transduced cells. The second generation of rAAV, known as self-complementary AAV (scAAV), was generated by deleting the terminal resolution site (TRS) of one ITR to produce a double-stranded DNA genome. Self-complementary AAV vectors bypass the rate-limiting step of converting the single-stranded genome to a double-stranded genome during rAAV transduction. Although self-complementary AAVs have improved transduction efficiencies in vitro and in vivo, the packaging capacity of self-complementary AAVs is effectively halved (approximately 2.5 kb).