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Record 1,  Textual support,  English

Record number: 1, Textual support number: 1 CONT

For gene therapy to realize its clinical potential, there must be efficient and safe strategies of delivering therapeutic genes to somatic cells in vivo. Perhaps this problem simply represents a special case of drug delivery in which the therapeutic gene constitutes the drug. But the relevance of traditional drug development to technological developments in gene therapy is questionable. Recombinant genes that can independently express a therapeutic RNA or protein are extraordinarily large and polar molecules -- 10,000 times larger than traditional pharmaceutical agents. Moreover, the administration of genes as therapy requires vehicles -- so-called vectors -- that encapsulate the gene and guide it to the target cell. The binding of the gene to the cell, its internalization, the transport of the administered genome to the nucleus, and the expression of the gene all constitute potential limitations of this process.  1, record 1, English, - therapeutic%20gene

Record 1,  Textual support,  French

Record number: 1, Textual support number: 1 CONT

L'un des aspect essentiels du transfert de gènes à visée thérapeutique est la vectorisation de l'ADN par un transporteur jusqu'à la cellule cible [...] dans la plupart des cas, l'ADN doit être introduit dans la cellule, soit à l'aide d'une technique physique ou chimique fragilisant les membranes cellulaires, soit combiné à un vecteur.  1, record 1, French, - g%C3%A8ne%20%C3%A0%20vis%C3%A9e%20th%C3%A9rapeutique

Record 1,  Textual support,  Spanish

Record 2,  Textual support,  English

Record number: 2, Textual support number: 1 CONT

For gene therapy to realize its clinical potential, there must be efficient and safe strategies of delivering therapeutic genes to somatic cells in vivo. Perhaps this problem simply represents a special case of drug delivery in which the therapeutic gene constitutes the drug. But the relevance of traditional drug development to technological developments in gene therapy is questionable. Recombinant genes that can independently express a therapeutic RNA or protein are extraordinarily large and polar molecules -- 10,000 times larger than traditional pharmaceutical agents. Moreover, the administration of genes as therapy requires vehicles -- so-called vectors -- that encapsulate the gene and guide it to the target cell. The binding of the gene to the cell, its internalization, the transport of the administered genome to the nucleus, and the expression of the gene all constitute potential limitations of this process.  1, record 2, English, - therapeutic%20gene

Record 2,  Textual support,  French

Record number: 2, Textual support number: 1 CONT

Mais surtout, à côté de la pathologie, un fantastique domaine s'ouvre au transfert et à la thérapie génique, c'est celui du gène considéré comme médicament. La médecine utilise en effet toute une série de molécules d'origine protéique, comme les facteurs antihémophilliques, l'insuline, l'hormone de croissance, pour ne citer que les plus importantes. Le futur pourrait être la fabrication directe par l'organisme des molécules actives après transfert de gènes dans les cellules permissives. Mieux encore, on pourra vraisemblablement dams l'avenir injecter directement par voie intramusculaire par exemple, l'ADN génique, les cellules de l'organisme se comportant ensuite comme de mini-usines de fabrication et de mini-unités de distribution.  1, record 2, French, - g%C3%A8ne%20consid%C3%A9r%C3%A9%20comme%20m%C3%A9dicament

Record number: 2, Textual support number: 2 CONT

Ceci ne saurait s'effectuer en dehors d'un contexte réglementaire adapté à la complexité des différents niveaux d'intervention que nécessite le transfert de gènes à visée thérapeutique.  1, record 2, French, - g%C3%A8ne%20consid%C3%A9r%C3%A9%20comme%20m%C3%A9dicament

Record 2,  Textual support,  Spanish

Record 3,  Textual support,  English

Record number: 3, Textual support number: 1 CONT

In the ideal world, the diseases would be cured for life by one treatment, with no side effects. And gene insertion into a chromosome in a target somatic cell would be site specific; in what is called homologous recombination, the healthy, or "therapeutic," gene would exactly replace the damaged copy  1, record 3, English, - therapeutic%20gene

Record 3,  Textual support,  French

Record number: 3, Textual support number: 1 CONT

Des gènes marqueurs aux gènes thérapeutiques. De nombreux participants ont manifesté la volonté d'utiliser des approches de marquage avant d'aborder le problème du transfert génique en vue d'une action thérapeutique.  2, record 3, French, - g%C3%A8ne%20th%C3%A9rapeutique

Record 3,  Textual support,  Spanish