Compounds and their use for specific and simultaneous...

C - Chemistry – Metallurgy – 12 – N

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C12N 15/115 (2010.01) A61K 31/475 (2006.01) A61K 31/7088 (2006.01) A61K 47/48 (2006.01) C07H 21/00 (2006.01) C12N 15/11 (2006.01) C12N 9/90 (2006.01)

Patent

CA 2519457

The invention relate to the use of a compound of formula A-B-C Wherein A is a DNA sequence-specific ligand capable of simultaneously and specifically recognizing a sequence common to genes of pathological interest; B is a linker arm, said linker arm being bound to the 3' end of A; C is a topoisomerase I posion; for the preparation of a drug for the treatment of a disease brought about by the expression of a gene and said gene is inhibited by the stabilized topoisomerase I-mediated DNA cleavage. Application, particularly, for the treatment of infective microorganism or virus, dismetabolic disease and autoimmune disease.

L'invention concerne l'utilisation d'un composé de formule A-B-C, dans laquelle A est un ligand spécifique de séquence ADN apte à reconnaître simultanément et de manière spécifique une séquence commune aux gènes d'intérêt pathologique ; B est un bras lieur, ledit bras lieur étant lié à la terminaison 3' de A ; C est un poison de topoisomérase I, pour préparer un médicament intervenant dans le traitement d'une pathologie provoquée par l'expression d'un gène et ledit gène est inhibé par la restriction de l'ADN induite par topoisomérase I stabilisée. L'invention s'applique en particulier au domaine du traitement de micro-organismes infectieux ou de virus, de pathologies dismétaboliques et de maladies auto-immunes.

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