AAV6-Syn-RFP

Viral vectors are widely used for gene delivery and manipulation of gene expression within the central nervous system. Among them, adeno-associated virus (AAV) has become increasingly popular due to its non-pathogenicity, lack of innate immune response, ability to effectively transduce dividing and non-dividing cells, and long-lasting gene expression. The effectiveness of AAV constructs in basic and pre-translational research has led to their successful introduction into clinical practice, including applications in the central nervous system. In the past decade, recombinant AAV has been used for gene delivery in clinical trials targeting brain, spinal cord, and retinal tissues. Seven products have been marketed, and there are more than 200 clinical trials using recombinant AAV (rAAV) worldwide.

Adeno-associated virus (AAV) is a small, non-enveloped virus belonging to the Parvoviridae family. It was discovered in the 1960s. rAAV consists of an icosahedral protein capsid and a recombinant DNA (up to 4.7 kb in the case of a single strand). The capsid contains three subunit proteins, VP1, VP2, and VP3, with a stoichiometric ratio of typically 5:5:50, and each capsid has 60 subunits. Multiple serotypes have been identified and observed to exhibit different tissue tropisms, making AAV a more attractive modality for gene therapy.