Winston Stephen M, Wiggins Kristin B, Schultz-Cherry Stacey, Davidoff Andrew M
Department of Surgery, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
Department of Host-Microbe Interactions, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
J Virol. 2025 Aug 19;99(8):e0073025. doi: 10.1128/jvi.00730-25. Epub 2025 Jul 14.
Adeno-associated virus (AAV) has proven its clinical efficacy in the realm of gene therapy, resulting in seven FDA-approved gene therapies. While AAV gene transfer research has predominantly focused on its utility in monogenic disorders, AAV vectors have been used as a platform for vaccines in over 50 preclinical studies over the last 25 years. Recombinant AAV-based vaccines have demonstrated induction and durability of antigen-specific adaptive immune responses in a variety of preclinical models. This mini-review serves as a comprehensive discussion of the basics of vaccine vector design and experimental considerations, highlighting engineering efforts to improve AAV vaccine efficacy, along with the known advantages and disadvantages of AAV-based vaccines from published pre-clinical studies.
腺相关病毒（AAV）已在基因治疗领域证明了其临床疗效，已有七种基因疗法获得美国食品药品监督管理局（FDA）批准。虽然AAV基因转移研究主要集中在其在单基因疾病中的应用，但在过去25年中，AAV载体已在50多项临床前研究中被用作疫苗平台。基于重组AAV的疫苗在多种临床前模型中已证明可诱导抗原特异性适应性免疫反应并具有持久性。本综述全面讨论了疫苗载体设计的基础和实验考虑因素，重点介绍了提高AAV疫苗疗效的工程学努力，以及已发表的临床前研究中基于AAV的疫苗的已知优缺点。
