OVERVIEW OF WAYS TO DELIVER GENETIC CONSTRUCTS TO CELLULAR TARGETS
Gene therapy (GT) is a new direction in medicine that uses nucleic acids (NA) as a therapeutic agent. GT can be used in the treatment of both hereditary diseases (spinal muscular atrophy, Duchenne myodystrophy) and diseases of mixed etiology (cancer, cardiovascular diseases, neurodegenerative) and acquired (for example, infectious) diseases. The main strategies of GT include gene replacement therapy, effects on gene expression, and suicidal gene therapy. There are a number of difficulties in the delivery of therapeutic NA, since free NA molecules are not stable and cannot independently penetrate into target cells. The development of safe and effective delivery methods is one of the key tasks of GT. Virus-based delivery systems are the most common due to their high efficiency, but they have a number of undesirable effects, which limits their use. The purpose of this article is to review alternative ways of delivering genetic constructs: using natural and artificial polymers, liposomes, peptides.
Authors
- Arina V. Ilina
- N. A. Krylova
- Haya Al-Hilal
- Dmitry O. Vinokhodov
- R. G. Sakhabeev
Publication Details
- Journal
- Russian Journal of Biotechnology
- Published
- 2025-01-01
- DOI
- https://doi.org/10.36807/3034-6738-2025-01-11-21
- Primary Topic
- Viral Infectious Diseases and Gene Expression in Insects
- Type
- article
- Field-Weighted Citation Impact
- 0.00