AAV DJ-Cre-GFP

AAV is an ideal candidate for gene therapy due to its low immunogenicity, limited neutralizing antibody production, and replication defects. AAV production requires cytopathogenicity, which occurs only after co-infection with a helper adenovirus or herpes virus. Intracranial interferon-β (IFN-β) gene therapy based on local administration of AAV vectors has been reported to be successful in non-invasive orthotopic glioblastoma models and is also effective against migratory tumors. AAV vectors have several key properties that make them useful for gene delivery in cancer therapy.

Early studies used capsids and viral machinery derived from AAV serotype 2 (AAV2). AAV2 remains the basis for most AAV systems, but engineered capsids with tissue-specific tropism or increased infectivity are now available, including DJ and DJ8. DJ serotypes have also been shown to effectively transfect many cultured cells, making DJ particularly suitable for cell culture and in vivo applications. Furthermore, specific promoters, the Cre/flox system, and gene editing via CRISPR make AAV cell-specific and allow new approaches in gene therapy and animal studies. Most protocols recommend purifying AAV from lysates of producer cells, which are grown in large cell stacks or cell culture factories to obtain enough AAV for animal experiments.