Huntingtin-related therapeutics and bioassay

Details Huntingtin-related therapeutics and bioassay Huntingtin-related therapeutics and bioassay

C - Chemistry, Metallurgy

12

Q

C12Q 1/00 (2006.01) C12Q 1/02 (2006.01) G01N 33/68 (2006.01)

Patent

CA 2271936

A method of identifying a compound useful in the treatment of a disease characterized by the presence of mutant huntingtin by: (a) providing a cell which expresses mutant huntingtin; (b) contacting the cell with a test compound; and (c) determining whether the level of clathrin-mediated endocytosis is increased in the presence of the test compound, compared to the level in the absence of the test compound. An increase in clathrin-mediated endocytosis is an indication that the test compound is useful in treating the disease.

La présente invention concerne un procédé d'identification d'un composé utilisé dans le traitement d'une maladie impliquant la présence d'une huntingtine mutante, ledit procédé consistant à: (a) fournir une cellule exprimant une huntingtine mutante; (b) à mettre en contact ladite cellule avec un composé test; et (c) à déterminer si le taux d'endocytose à médiation clathrine a augmenté en présence de ce composé test par rapport au même taux en l'absence du composé d'essai. Une augmentation de l'endocytose à médiation clathrine indique que le composé test peut être utilisé dans le traitement de la maladie.

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