[Gene therapy of monogenic hereditary diseases. Duchenne myodystrophy]

The paper highlights the new trends in gene therapy research area and clinical trials. It should be noted that the majority of firms involved in development of the scientific approaches to gene therapy are concentrated in the United States. The investments of the given companies on development and research of new genetic constructs also delivery systems make hundred millions dollars. The greatest part (more than 80%) of gene therapy clinical trials projects are also connected with the US research departments; the majority of them is related to tumor therapy. The advantages and drawbacks of the main methods of nucleic acids delivery to the cells are considered; diseases that are attempted to be treated using gene therapy methods are listed. A special attention of the review is devoted to the modern stand in research on cell and Duchenne muscular dystrophy (DMD) gene therapy, also brief description of basic results achieved in the authors laboratory is given. Basic original results of transfection of mdx mice (DMD biological models) with dystrophin cDNA delivered by gene gun, cationic liposomes, synthetic microspheres, viral olygopeptides and lactoferrine are summarized.