Abstract <p>This review contains up-to-date information about existing methods of gene therapy (GT) and GT-products approved for use in the world and Russian Federation, as well as those currently at the stage of development and clinical trials. The stages of GT-drug development as well as methods for targeted delivery of genetic material into cells and tissues are considered. Particular attention is paid to the characteristics of viral vectors and some methods of their design and targeting. Approaches for modifying the sensitivity of viruses to endogenous (pH, redox potential, enzyme content) and exogenous (light, magnetic field, temperature) stimuli are discussed, as are various methods used for this purpose, such as pseudotyping, incorporation of a targeting ligand into the viral genome, and physical exposure. The various adapters are also described in detail. All of these modification methods are based on rational design or directed evolution approaches. Fundamental research in this field of science is aimed at developing new strategies for the treatment of various diseases, including those still considered incurable.</p>

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Stimulus-Responsive Delivery Systems for Gene Therapy Products

  • N. G. Sakhno,
  • O. V. Gunar

摘要

Abstract

This review contains up-to-date information about existing methods of gene therapy (GT) and GT-products approved for use in the world and Russian Federation, as well as those currently at the stage of development and clinical trials. The stages of GT-drug development as well as methods for targeted delivery of genetic material into cells and tissues are considered. Particular attention is paid to the characteristics of viral vectors and some methods of their design and targeting. Approaches for modifying the sensitivity of viruses to endogenous (pH, redox potential, enzyme content) and exogenous (light, magnetic field, temperature) stimuli are discussed, as are various methods used for this purpose, such as pseudotyping, incorporation of a targeting ligand into the viral genome, and physical exposure. The various adapters are also described in detail. All of these modification methods are based on rational design or directed evolution approaches. Fundamental research in this field of science is aimed at developing new strategies for the treatment of various diseases, including those still considered incurable.