Syn-FLEX-jGCaMP7c AAV (Serotype 9)

Gene therapy uses nucleic acids as therapeutic agents to permanently correct a disease. The success of central nervous system (CNS) gene therapy approaches depends largely on the chosen delivery system. Non-viral vectors (e.g., liposomes, exosomes, polymer nanoparticles) are considered a promising option due to their simple and cost-effective production methods and safety. However, their efficiency is relatively low, and the mediated effects are transient, requiring repeated administration and carrying the potential risk of eliciting an immune response. Therefore, recombinant viral vectors are considered the most effective system to achieve long-term stable gene expression in the CNS.

AAV vectors have become the vector of choice for CNS gene transfer. Although both have limited packaging capacity compared to adenovirus and herpes simplex virus, they have significant advantages, including persistent gene expression and lack of toxicity. As a result, extensive preclinical studies have successfully demonstrated the therapeutic potential of this vector in neurological diseases. AAV has important advantages, including a better safety profile due to its wild-type non-pathogenicity. Furthermore, AAV generally induces higher transgene expression levels than LV, as well as a greater range of vector spread following direct infusion into the brain (1–3 mm vs. 500–700 μm). In summary, AAV mediates safe, widespread, and stable transgene expression and is therefore the most common vector used in clinical studies of CNS disorders.