AAV DJ/8-CAG-GFP

Since the first discovery of adeno-associated virus (AAV) in 1965, hundreds of variants have been isolated from adenovirus libraries or primate tissues. Different serotypes of AAV1-10 can preferentially transduce or induce specific cell or tissue types, thereby achieving organ-based gene delivery. Various recombinant AAVs (rAAVs) have been constructed to transduce various living cells (dividing and non-dividing) and deliver target genes. rAAV contains a 20-25 nm non-enveloped capsid (virion protein 1 [VP1], VP2, and VP3) and a single-stranded DNA (ssDNA) genome of < 4.9 kb, including the target gene, flanked by inverted terminal repeats. The Kay, Samulski, and Schaffer laboratories constructed novel engineered chimeric AAV capsids, such as AAV-DJ, -DJ8, etc., to evade the neutralization of AAV by pre-existing serum antibodies, improve in vivo infection efficiency, and enhance circulation stability.

Compared with traditional nanoparticle-mediated gene delivery vectors, rAAV has the advantages of high infection rate, long-term transgene expression, low immunogenicity and minimal toxicity in clinical applications. In the past two decades, more than 130 AAV-delivered gene therapies have been evaluated in clinical trials, and hundreds of clinical trials are ongoing for the treatment of Alzheimer's disease, Parkinson's disease and other diseases.