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AUTOPHAGY MODULATORS FOR TREATING NEURODEGENERATIVE DISEASES

机译:用于治疗神经退行性疾病的自噬调节剂

摘要

Disclosed is a method of decreasing an amount of protein aggregate in a cell comprising contacting the cell with a compound of the formula (I): wherein R1, R2, and R3 are as defined herein, in an amount sufficient to induce autophagy in the cell and cause the degradation of the protein aggregate within the cell. Also disclosed is a method of treating or preventing a condition, syndrome, or disease in a mammal in need thereof, wherein the condition, syndrome, or disease is selected from Huntington's disease, Alzheimer's disease, Parkinson's disease, Hutchinson-Gilford progeria syndrome, Niemann-Pick type C disease, spinobulbar muscular atrophy (Kennedy disease), dentatorubral-pallidoluysian atrophy (Haw-River syndrome), spinocerebellar ataxia type 1, spinocerebellar ataxia type 2, spinocerebellar ataxia type 3, spinocerebellar ataxia type 6, and spinocerebellar ataxia type 7, comprising administering to the mammal the compound.
机译:公开了一种减少细胞中蛋白质聚集体量的方法,该方法包括使细胞与式(I)的化合物接触:其中R 1 ,R 2 和R 3 如本文所定义,其量足以在细胞中诱导自噬并引起细胞内蛋白质聚集体的降解。还公开了在需要其的哺乳动物中治疗或预防病症,综合症或疾病的方法,其中所述病症,综合症或疾病选自亨廷顿氏病,阿尔茨海默氏病,帕金森氏病,哈钦森-吉尔福德早衰综合症,尼曼-C型疾病,脊椎小球性肌萎缩症(肯尼迪病),齿龈-睑板肌萎缩症(Haw-River综合征),脊髓小脑性共济失调1型,脊髓小脑性共济失调2型,脊髓小脑性共济失调3型,脊髓小脑性共济失调6型和脊髓小脑性失调7型,包括将所述化合物给予哺乳动物。

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