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

Journal: 3 Biotech
Published Date: Jul 1, 2025

Abstract

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.

Authors

  • Kuldeep Singh
    Department of Electronics Technology, Guru Nanak Dev University, Amritsar, India. kuldeep.ece@gndu.ac.in.
  • Divya Jain
    Department of Microbiology, School of Applied and Life Sciences, Uttaranchal University, Dehradun, Uttarakhand India.
  • Pranshul Sethi
    Department of Pharmacology, College of Pharmacy, Shri Venkateshwara University, Gajraula, UP 244236 India.
  • Jeetendra Kumar Gupta
    Department of Pharmacology, Institute of Pharmaceutical Research, GLA University, Mathura, Uttar Pradesh India.
  • Anubhav Dubey
    Department of Pharmacology, Maharana Pratap College of Pharmacy, Kanpur, Uttar Pradesh India.
  • Abdullah Al Noman
    School of Pharmacy, BRAC University, Dhaka, Bangladesh.
  • Shipra Lal
    IES Institute of Pharmacy, IES University, Bhopal, Madhya Pradesh 462044 India.
  • Pranab Dev Sharma
    Biotechnology Program, Department of Mathematics and Natural Science, BRAC University, Dhaka, Bangladesh.
  • Emad M Abdallah
    Department of Biology, College of Science, Qassim University, 51452 Qassim, Saudi Arabia.

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