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Strategies for gene therapy


Hématologie. Volume 2, Number 1, 79-93, Janvier - Février 1996, THERAPEUTIQUE BIOTECHNOLOGIQUE

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Author(s) : Yves Beuzard

Summary : Whatever may be the pathology to be cured, infectious, leukemic, tumoral, genetic or degenerative, the stratgy for the transfer of a marker or therapeutic genes requires a comprehensive control of five parameters : 1) the gene of interest (transgene) ; 2) the transfer system ; 3) the target cell ; 4) the in vivo evaluation of the therapy and 5) the safety. For a specific disease, the therapeutic goals for gene transfer and the methods to reach them are multiple, even in the case of a monogenic disease. The pratical treatments we can forsee will be diverse, from the simple injection for vaccination of infectious diseases to sophisticated gene therapy requiring specific facilities and some time confinement of patients. An overview of the present strategies and their evolutions during the last fifteen years will be presented, from myth to reality.

Keywords : gene therapy, genetics, cancer, cardiovascular diseases, hematology, hemoglobinopathies, AIDS.

 

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