Viral Vector-Based Gene Therapy

Int J Mol Sci. 2023 Apr 23;24(9):7736. doi: 10.3390/ijms24097736.


Gene therapy is a technique involving the modification of an individual's genes for treating a particular disease. The key to effective gene therapy is an efficient carrier delivery system. Viral vectors that have been artificially modified to lose their pathogenicity are used widely as a delivery system, with the key advantages of their natural high transduction efficiency and stable expression. With decades of development, viral vector-based gene therapies have achieved promising clinical outcomes. Currently, the three key vector strategies are based on adeno-associated viruses, adenoviruses, and lentiviruses. However, certain challenges, such as immunotoxicity and "off-target", continue to exist. In the present review, the above three viral vectors are discussed along with their respective therapeutic applications. In addition, the major translational challenges encountered in viral vector-based gene therapies are summarized, and the possible strategies to address these challenges are also discussed.

Keywords: adeno-associated viruses; adenoviruses; challenges; gene therapy; lentiviruses.

Publication types

  • Review

MeSH terms

  • Adenoviridae / genetics
  • Dependovirus / genetics
  • Gene Transfer Techniques
  • Genetic Therapy* / methods
  • Genetic Vectors* / genetics
  • Lentivirus / genetics

Grants and funding

This research received no external funding.