Gene-therapeutic process using antibiotic-resistance...

C - Chemistry – Metallurgy – 12 – N

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C12N 15/85 (2006.01) A61K 48/00 (2006.01) C12N 15/12 (2006.01)

Patent

CA 2186242

Use of a vector DNA for the production of a pharmaceutical agent for the treatment of mammals or humans by gene therapy in which the vector DNA causes a modulation, correction or activation of the expression of an endogenous gene or the expression of a gene introduced into the cells of the mammal or human by the vector DNA, which is characterized in that the vector nucleic acid does not contain an active antibiotic resistance gene or one which is relevant for humans. Such a vector DNA is particularly suitable for treatment of the respiratory tract, the intestinal tract and the skin by gene therapy.

L'invention concerne l'utilisation d'un ADN vectoriel pour préparer un médicament de traitement par thérapie génique de mammifères ou d'humains. L'ADN vectoriel provoque une modulation, une correction ou une activation de l'expression d'un gène endogène ou l'expression d'un gène introduit par l'ADN vectoriel dans les cellules du mammifère ou de l'homme concerné. L'invention se caractérise en ce que l'acide nucléique vectoriel ne contient pas de gène d'antibiorésistance actif ou significatif pour l'homme. Les ADN vectoriels de ce type conviennent particulièrement au traitement par thérapie génique des voies respiratoires, du tube digestif et de la peau.

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