The ultimate goal in the management of inherited as well as acquired diseases is a rational therapy with the aim to eliminate the underlying biochemical defects, rather than a symptomatic treatment.
Among other approaches somatic gene therapy is a promising candidate to meet these objectives and appears to have the potential to revolutionize modern medicine.
Gene therapy is characterized by the transfer of genetic information to a patient through the use of recombinant DNA technology.
Several strategies for the treatment of monogenetic disorders as well as chronical diseases like cancer and AIDS have been used in various somatic gene therapy projects.
So far, 329 clinical studies (phases I, I/II and II) with over 2500 patients have been initiated worldwide since 1989.
No significant toxicity and adverse side effects have been observed.
To allow efficient transfer of the therapeutic genes, a variety of gene delivery techniques have been developed based on viral and non-viral vector systems.
For the success of this technology it is vital to achieve regulated and sustained expression of foreign genes in specific target tissues.
This will be crucial for the widespread application of somatic gene therapy.
So far none fo the gene delivery systems is able to meet the requirements of safety, efficiency and specificity demonstrating that vector research will be an important focus in the development of optimized transfer methods. (...)
Mots-clés Pascal : Thérapie génique, Cellule somatique, Gène, Article synthèse, Recherche développement, DNA recombinant, Homme, Toxicité, Effet secondaire, Législation, Réglementation, Biotechnologie
Mots-clés Pascal anglais : Gene therapy, Somatic cell, Gene, Review, Research and development, Recombinant DNA, Human, Toxicity, Secondary effect, Legislation, Regulation, Biotechnology
Notice produite par :
Inist-CNRS - Institut de l'Information Scientifique et Technique
Cote : 99-0044703
Code Inist : 002A31D01D. Création : 31/05/1999.