Molecular targets and compounds, and methods to identify the...

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G01N 33/68 (2006.01) C07K 14/47 (2006.01)

Patent

CA 2711585

The present invention relates to methods and assays for identifying agents capable of inhibiting the mutant hunt-ingtin protein, inhibiting or reducing polyglutamine-induced protein aggregation, and/or altering huntingtin protein conformation, which inhibition is useful in the prevention, amelioration and / or treatment of neurodegenerative diseases, and protein aggrega-tion diseases more generally. In particular, the present invention provides methods and assays for identifying agents for use in the prevention and / or treatment of Huntingtons disease. The invention provides polypeptide and nucleic acid TARGETs and siRNA sequences based on these TARGETs.

Cette invention concerne des procédés et des dosages permettant didentifier des agents capables dinhiber la protéine huntingtine mutante, dinhiber ou de réduire lagrégation protéinique induite par la polyglutamine, et/ou à modifier la conformation de la protéine huntingtine, ladite inhibition étant utile dans la prévention, lamélioration et/ou le traitement des maladies neurodégénératives, et plus généralement des maladies à agrégation protéinique. Linvention concerne en particulier des procédés et des dosages permettant didentifier des agents à utiliser dans la prévention et/ou le traitement de la maladie de Huntington. Linvention concerne des cibles polypeptidiques et dacides nucléiques, ainsi que des séquences de siRNA basées sur ces CIBLES.

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