Voprosy meditsinskoi khimii (ISSN 0042-8809)

Gene therapy of monogenic hereditary diseases. Duchene miodistrophy


1. D.O. Ott Institute of Obstetrics and Gynaecology, Russian Academy of Medical Sciences
PubMed Id: 11033887
Year: 2000 vol: 46  issue:3  pages: 279-292
Abstract: The paperhighlights the new trends in gene therapy research area and clinical trials. It should benoted that the majority of firms involved in development of the scientific approaches togene therapy are concentrated in the United States. The investments of the given companieson development and research of new genetic constructs also delivery systems make hundredmillions dollars. The greatest part (more than 80 %) of gene therapy clinical trialsprojects are also connected with the US research departments; the majority of them isrelated to tumor therapy. The advantages and drawbacks of the main methods of nucleicacids delivery to the cells are considered; diseases that are attempted to be treatedusing gene therapy methods are listed. A special attention of the review is devoted to themodern stand in research on cell and Duchenne muscular dystropy (DMD) gene therapy, alsobrief description of basic results achieved in the authors laboratory is given. Basicoriginal results of transfection of mdx mice (DMD biological models) with dystrophin cDNAdelivered by gene gun, cationic liposomes, synthetic microspheres, viral olygopeptides andlactoferrine are summarized.
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Reference: Baranov V.S., Baranov A.N., Gene therapy of monogenic hereditary diseases. Duchene miodistrophy, Voprosy meditsinskoi khimii, 2000, vol: 46(3), 279-292.