AAV6-CAG-Cre-2A-GFP

Adeno-associated virus (AAV) is a non-pathogenic parvovirus that has become one of the most popular in vivo gene transfer tools for gene therapy. AAV vectors can transduce many different cell types in vivo, resulting in stable transgene expression in post-mitotic tissues. AAV vector particles consist of a recombinant DNA genome (vector genome), which is usually single-stranded (ss), packaged into an 18- to 25-nm capsid containing 60 capsid monomers arranged in a T = 1 icosahedral symmetry. Capsids can be derived from several naturally occurring AAV serotypes, or from engineered capsids. Recent clinical trials with AAV vectors have not only demonstrated the safety of the vectors for intravenous and topical vector administration, but also therapeutic efficacy. The transduction efficiency of AAV depends on the successful completion of a multistep infection process. After binding to cellular receptors, AAV particles are internalized and transported to the nucleus. The AAV particle then enters the nucleus, where the ssDNA genome is released from the capsid, a process called uncoating, and converted into a double-stranded (ds) DNA molecule. It is therefore conceivable to state that the efficiency of cell transduction is at least dependent on the efficiency of vector internalization, including nuclear delivery and the efficiency of uncoating in the nucleus.