Method of screening compounds

C - Chemistry – Metallurgy – 12 – N

Patent

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Details

C12N 15/63 (2006.01) A01K 67/027 (2006.01) A01K 67/033 (2006.01) A61K 31/10 (2006.01) A61K 31/167 (2006.01) A61K 31/175 (2006.01) A61K 31/196 (2006.01) A61K 31/197 (2006.01) A61K 31/216 (2006.01) A61K 31/255 (2006.01) A61K 31/352 (2006.01) A61K 31/366 (2006.01) A61K 31/44 (2006.01) C12N 15/85 (2006.01) C12N 15/87 (2006.01) C12P 21/00 (2006.01) G01N 33/50 (2006.01)

Patent

CA 2470311

The present invention is directed to a novel, target-blind approach to drug discovery. The concept is to model human phenotypes in a teleost, such as a zebrafish, and then screen compounds, e.g., small molecules, for their ability to alter the phenotype. Because the screen is performed with a whole vertebrate organism and uses a phenotype as the output, the need to first identify target genes is eliminated. This approach is powerful because a single screen can theoretically detect drugs affecting any target relevant to the phenotype being observed, even if those targets are not yet characterized.

La présente invention se rapporte à une nouvelle approche sans ciblage préalable permettant de découvrir des médicaments. A cet effet, on simule des phénotypes humains chez un poisson téléostéen, tel qu'un poisson zèbre, puis on crible des composés, notamment de petites molécules, pour leur aptitude à modifier le phénotype. L'identification préalable de gènes cibles s'avère inutile puisque le criblage s'effectue avec un organisme vertébré complet et utilise un phénotype comme données de sortie. Cette approche est puissante, car un seul criblage peut en théorie détecter des médicaments affectant une cible quelconque ayant rapport au phénotype en cours d'observation, même si ces cibles n'ont pas encore été caractérisées.

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