Voprosy meditsinskoi khimii (ISSN 0042-8809)

Gene therapy of hereditary diseases


1. Research Centre for Medical Genetics, Russian Academy of Medical Sciences
PubMed Id: 11033886
Year: 2000 vol: 46  issue:3  pages: 264-278
Abstract: In the review the main advantages in development of the approaches togene therapy of hereditary diseases are presented. Now more than 1000 genes of hereditarydiseases are mapped and some hundreds are cloned which is prerequisite for gene therapy.The transfer of the recombinant gene into the cell and the subsequent expression of thetransgene product are the rate-limiting steps for successful gene therapy. A variety ofmethods, including the use of physical methods, modified viruses and synthetic vectors,are currently being used in experiments and clinical trials. Since the approval andinitiation of the first human gene therapy trial to treat ADA deficiency, there have beenseveral dozen approved gene therapy trials but clear clinical result was stated for ADAdeficiency only.
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Reference: Ginter E.K., Gene therapy of hereditary diseases, Voprosy meditsinskoi khimii, 2000, vol: 46(3), 264-278.