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<p>In recent years, adeno-associated viruses (AAVs) have emerged as leading vectors in gene therapy, with several FDA-approved treatments and ongoing clinical trials demonstrating their effectiveness in treating inherited retinal diseases, hemophilia, and Duchenne muscular dystrophy, among others. However, AAV-based therapies still face challenges, including immune responses and side effects, due to high viral doses. To address these challenges, various strategies have been developed, such as creating new viral capsids, optimizing gene expression regulation, and improving delivery methods. Localized delivery is a promising direction, utilizing the tissue tropism of AAVs to reduce systemic side effects and lower the required viral dose, thus improving targeting and efficiency, especially for organs that are difficult to treat with conventional methods. These innovations have opened new pathways for the clinical application of AAVs. This review aims to provide a comprehensive summary of the various applications of AAVs, offer valuable insights for future research directions, and holds significant importance for researchers and clinicians in the field. As AAV therapy continues to evolve, this article emphasizes its transformative potential in treating genetic diseases, indicating the central role of AAV in the future of gene therapy.</p>

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Advances and Challenges in Adeno-Associated Virus Gene Therapy Applications of Localized Delivery Strategies

  • 期刊论文

  • 发表日期：

    2025年7月15日

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  • Jin-hao Chen,
  • Li-jun Zhan,
  • Cun-ming Lv,
  • Jun-bo Teng,
  • Chun-yu Cao

摘要

In recent years, adeno-associated viruses (AAVs) have emerged as leading vectors in gene therapy, with several FDA-approved treatments and ongoing clinical trials demonstrating their effectiveness in treating inherited retinal diseases, hemophilia, and Duchenne muscular dystrophy, among others. However, AAV-based therapies still face challenges, including immune responses and side effects, due to high viral doses. To address these challenges, various strategies have been developed, such as creating new viral capsids, optimizing gene expression regulation, and improving delivery methods. Localized delivery is a promising direction, utilizing the tissue tropism of AAVs to reduce systemic side effects and lower the required viral dose, thus improving targeting and efficiency, especially for organs that are difficult to treat with conventional methods. These innovations have opened new pathways for the clinical application of AAVs. This review aims to provide a comprehensive summary of the various applications of AAVs, offer valuable insights for future research directions, and holds significant importance for researchers and clinicians in the field. As AAV therapy continues to evolve, this article emphasizes its transformative potential in treating genetic diseases, indicating the central role of AAV in the future of gene therapy.