Target sequences and methods to identify the same, useful in...

G - Physics – 01 – N

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G01N 33/50 (2006.01)

Patent

CA 2711587

The present invention relates to methods and assays for identifying agents capable of inhibiting the mutant hunt-ingtin protein, inhibiting or reducing cell death, in particular cell death associated with polyglutamine-induced protein aggrega-tion, which inhibition is useful in the prevention, amelioration and / or treatment of neurodegenerative diseases, and Huntington's disease more generally. In particular, the present invention provides methods and assays for identifying agents for use in the pre-vention and / or treatment of Huntingtons disease. The invention provides polypeptide and nucleic acid TARGETs and siRNA se-quences based on these TARGETS.

La présente invention concerne des procédés et des dosages pour identifier des agents capables dinhiber la protéine huntingtine mutante, dinhiber ou de réduire la mort cellulaire, en particulier la mort cellulaire associée à lagrégation de protéine induite par la polyglutamine, linhibition étant utile dans la prévention, lamélioration et/ou le traitement de maladies neurodégénératives et de la maladie de Huntington plus généralement. En particulier, la présente invention fournit des procédés et dosages pour identifier des agents destinés à être utilisés dans la prévention et/ou le traitement de la maladie de Huntington. Linvention fournit des cibles de polypeptide et dacide nucléique et des séquences dARNsi basées sur ces cibles.

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