AAV8-Syn-FLPo

The success of gene therapy relies on an efficient method to transfer the appropriate genetic material to the correct tissue. Adeno-associated virus (AAV) vectors have become a popular vector system for therapeutic gene transfer due to their non-pathogenic and broad-spectrum, low immunogenicity, and potential to achieve efficient and long-lived gene transfer. AAV is a non-enveloped, single-stranded DNA virus with a genome size of 4.7 kb and an icosahedral capsid of approximately 25 nm that was discovered in the 1960s as a contaminant in simian adenovirus preparations.

The AAV capsid consists of a total of 60 copies of the viral proteins VP1-3, with an average population ratio of 1:1:10. The biology of AAV has been extensively studied. A defining feature of AAV is its dependence on helper virus co-infection (such as adenovirus or herpes virus) for efficient replication. Replication-defective AAV virus-like particles (also called recombinant AAV [rAAV]), in which all viral open reading frames in the viral genome have been eliminated and contain heterologous genetic information, can be assembled and packaged into high vector yields for gene transfer applications.