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<p>Gene therapy offers a revolutionary approach for treating genetic and acquired disorders by delivering therapeutic genes to target cells. Viral vectors, engineered for efficient gene delivery, have become indispensable due to their high transduction efficiency, enhanced targeting specificity, and minimized immunogenicity. This review explores recent advancements in viral vector platforms, focusing on intracellular trafficking, transgene expression, and strategies to optimize vector design. Key challenges, including immune responses, insertional mutagenesis, and manufacturing scalability, are critically examined alongside innovative solutions such as hybrid delivery systems and AI-driven capsid design. The impact of these innovations is underscored by the 28 ongoing clinical trials and 16 FDA/EMA-approved gene therapies. In addition, integrating artificial intelligence for vector optimization and CRISPR-based gene correction holds great potential for precision medicine. Future directions include refining non-viral alternatives and improving biomanufacturing for enhanced accessibility. These advancements collectively drive the field toward safer and more effective gene therapy solutions.</p>

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Advances in viral vector-based delivery systems for gene therapy: a comprehensive review

  • 期刊论文

  • 发表日期：

    2025年5月30日

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  • Kuldeep Singh,
  • Divya Jain,
  • Pranshul Sethi,
  • Jeetendra Kumar Gupta,
  • Anubhav Dubey,
  • Abdullah Al Noman,
  • Shipra lal,
  • Pranab Dev Sharma,
  • Emad M. Abdallah

摘要

Gene therapy offers a revolutionary approach for treating genetic and acquired disorders by delivering therapeutic genes to target cells. Viral vectors, engineered for efficient gene delivery, have become indispensable due to their high transduction efficiency, enhanced targeting specificity, and minimized immunogenicity. This review explores recent advancements in viral vector platforms, focusing on intracellular trafficking, transgene expression, and strategies to optimize vector design. Key challenges, including immune responses, insertional mutagenesis, and manufacturing scalability, are critically examined alongside innovative solutions such as hybrid delivery systems and AI-driven capsid design. The impact of these innovations is underscored by the 28 ongoing clinical trials and 16 FDA/EMA-approved gene therapies. In addition, integrating artificial intelligence for vector optimization and CRISPR-based gene correction holds great potential for precision medicine. Future directions include refining non-viral alternatives and improving biomanufacturing for enhanced accessibility. These advancements collectively drive the field toward safer and more effective gene therapy solutions.