Adeno-associated virus as a delivery vector for gene therapy of human diseases | Signal Transduction and Targeted Therapy

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript. Advertisement Signal Transduction and Targeted Therapy volume  9, Article number: 78 (2024) Cite this article 18k Accesses 2 Citations 29 Altmetric Metrics details Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene expression in different tissues. Recombinant AAV (rAAV) has been engineered for enhanced specificity and developed as a tool for treating various diseases. However, as rAAV is being more widely used as a therapy, the increased demand has created challenges for the existing manufacturing methods.

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• Harshu Musunuri