Please read Dr. Maturana’s article in the Journal of Allergy and Clinical Immunology titled, “Persistent transgene expression in peripheral tissues one year post intravenous and intramuscular administration of AAV vectors containing the alphaherpesvirus latency-associated promoter 2.”
Gene delivery via recombinant adeno-associated virus (rAAV) stands as a cornerstone in both research and clinical applications due to its enduring transgene expression and safety profile. This aspect is essential for the efficacy and acceptance of AAV gene therapy as a reliable treatment option for various genetic and acquired diseases. Sustained expression ensures long-term therapeutic benefits, especially for chronic diseases that require persistent treatment. This reduces the need for frequent re-administration, improving patient compliance and reducing the healthcare burden. Besides, a good safety profile minimizes the risk of adverse effects, such as immune responses or off-target effects, which could compromise patient health or the success of the therapy. Ensuring safety is essential for gaining regulatory approval and public trust in gene therapy treatments. Despite these advantages, the challenge persists in precisely targeting specific tissues and cell types. While tissue-specific promoters exist, ubiquitous promoters offer robust and enduring transgene expression, potentially surpassing their specialized counterparts. To read the full article.
Persistent transgene expression in peripheral tissues one year post intravenous and intramuscular administration of AAV vectors containing the alphaherpesvirus latency-associated promoter 2. Maturana CJ, Engel EA. Front Virol. 2024;4:1379991. PMID: 38665693 PMCID: PMC11044866 DOI: 3389/fviro.2024.1379991