SMALL COMPOUNDS THAT CORRECT PROTEIN MISFOLDING AND USES THEREOF
    99.
    发明公开
    SMALL COMPOUNDS THAT CORRECT PROTEIN MISFOLDING AND USES THEREOF 审中-公开
    整改蛋白的小分子化合物及其用途错误折叠

    公开(公告)号:EP1909812A2

    公开(公告)日:2008-04-16

    申请号:EP06800453.0

    申请日:2006-07-27

    IPC分类号: A61K38/04

    摘要: The invention features compositions and methods comprising at least one compound selected from a proteasomal inhibitor, an autophagy inhibitor, a lysosomal inhibitor, an inhibitor of protein transport from the ER to the Golgi, an Hsp90 chaperone inhibitor, a heat shock response activator, a glycosidase inhibitor or histone deacetylase inhibitor that are useful for treating or preventing a protein conformation disease in a subject by correcting misfolded proteins in vivo. The compositions and methods can further comprise an 11-cis-retinal or 9-cis-retinal compound. Examples of protein conformation disorders and/or diseases may include retinitis pigmentosa, age-related macular degeneration, glaucoma, corneal dystrophies, retinoschises, Stargardt's disease, autosomal dominant druzen, Best's macular dystrophy, αl -antitrypsin deficiency, cystic fibrosis, Huntington's disease, Parkinson's disease, Alzheimer's disease, nephrogenic diabetes insipidus, cancer and/or Jacob-Creutzfeld disease.