C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/11 (2006.01) A61K 31/713 (2006.01) A61K 45/00 (2006.01) C07H 21/02 (2006.01) C07K 16/18 (2006.01) C12N 9/22 (2006.01) C12Q 1/02 (2006.01) C12Q 1/68 (2006.01) G01N 33/68 (2006.01)
Patent
CA 2653052
The present invention is directed to preventing the consequences of the misfolding of proteins, such as those associated with protein folding diseases. Provided are methods of treatment that involve administering an agent that decreases the level of the heat shock protein ATPase Aha1 and/or related molecules with similar function. Such methods can result in the rescue of folding, trafficking, and function of proteins with suboptimal folding kinetics. Also provided are screening methods to identify agents for the treatment of protein misfolding diseases.
La présente invention a pour but de prévenir les conséquences du mauvais repliement de protéines, telles celles qui sont associées aux maladies liées au repliement des protéines. L'invention concerne des méthodes de traitement qui consistent à administrer un agent diminuant le niveau de la protéine de choc thermique ATPase Aha 1 et/ou de molécules associées dotées d'une fonction analogue. De telles méthodes permettent de rétablir le repliement, le trafic et la fonction des protéines qui présentent une cinétique de repliement sous-optimale. L'invention se rapporte également à des procédés de criblage permettant d'identifier des agents destinés à traiter des maladies liées au repliement des protéines.
Balch William E.
Lapointe Paul G.
Venable John D.
Wang Xiaodong
Yates John R. III
Smart & Biggar
The Scripps Research Institute
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