公开(公告)号：EP2164865A2

公开(公告)日：2010-03-24

申请号：EP08759248.1

申请日：2008-06-13

申请人： Hochschule Mannheim ,  Max-Delbrück-Centrum für Molekulare Medizin (MDC)

发明人： KIOSCHIS-SCHNEIDER, Petra ,  HAFNER, Mathias ,  AMMERSCHLÄGER, Manuel ,  RITZ, Sandra ,  HOLLOSCHI, Andreas ,  WANKER, Erich, E.

IPC分类号： C07K14/47 ,  C12N15/12 ,  C12N5/10 ,  A61P25/00 ,  A61P35/00 ,  A61K31/00 ,  A61K31/352 ,  A61K31/58

CPC分类号： A61K31/00 ,  A61K31/352 ,  A61K31/58 ,  G01N33/6896 ,  G01N2500/10 ,  G01N2800/2835

摘要： The present invention relates to tetranortriterpenoid compounds and pharmaceutical compositions thereof, which are provided for use in the treatment, diagnosis and/or prevention of trinucleotide repeat disorders (like a polyglutamine diseases, e.g Huntingdon's disease), amyloid diseases, neurodegenerative disease, protein misfolding diseases or tumors. The tetranortriterpenoid compounds of the present invention are further provided for the reduction and/or inhibition of the aggregation of amyloidogenic proteins, preferably of polyglutamine proteins (such as huntingtin) as well as for increasing proteasome activity. The present invention furthermore relates to nucleic acids, comprising the nucleotide sequences of two huntingtin fragments, as well as to cells and kits, which are useful in methods for assessing the aggregation of huntingtin and in methods for identifying compounds, which modulate the aggregation of huntingtin.