DNA lipoplexes for gene therapy: a review

Authors

  • Rajashree Hirlekar Department of Pharmacy, Vivekanand Education Society’s College of Pharmacy, Chembur (E), Maharashtra, India
  • Revathi Mudaliyar Blumen Biovitals Healthkare Pvt Ltd, Mumbai, India
  • Samruddhi Nandkumar Borate Department of Pharmacy, Birla Institute of Technology and Science (BITS) Pilani, Hyderabad, India , Birla Institute of Technology and Science - Hyderabad Campus image/svg+xml
  • Prachiti Raut Department of Pharmacy, Vivekanand Education Society’s College of Pharmacy, Chembur (E), Maharashtra, India

DOI:

https://doi.org/10.1590/s2175-97902026e24428

Keywords:

Lipoplexes, Gene therapy, Non-viral vectors, Cationic liposomes, Cationic lipids

Abstract

Gene therapy holds substantial promise for the treatment of a wide range of genetic and acquired diseases. Despite its potential, efficient and safe delivery of therapeutic genes into target cells remains a significant challenge. Lipoplexes, lipid-base formulations complexed with nucleic acids, have emerged as an attractive non-viral vector for gene delivery due to their ability to encapsulate and protect nucleic acids and facilitate their delivery into cells. This review focuses on the design, mechanism of action, and structure of lipoplexes for gene therapy and discusses the role of various lipids used in the formation of lipoplexes. Furthermore, we explore the application of lipoplexes in various disease models, cystic fibrosis, and cancers, highlighting recent advances. We conclude that while challenges remain, lipoplexes represent a promising strategy for safe and effective gene therapy.

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2026-07-10

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DNA lipoplexes for gene therapy: a review. (2026). Brazilian Journal of Pharmaceutical Sciences, 62, e24428. https://doi.org/10.1590/s2175-97902026e24428