Use of indole-derived compounds for the preparation of a medicament that can be used to treat diseases related to the splicing process
申请人:Tazi Jamal
公开号:US20070054905A1
公开(公告)日:2007-03-08
The invention relates to indole-derived compounds and to the use of said compounds for the preparation of a medicament that can be used to treat diseases related to the process of splicing pre-messenger RNAs in the cell, such as Frasier syndrome, frontotemporal dementia linked to chromonsoem 17 (a form of Parkinson's disease), Leigh syndrome (a type of encephalopathy), atypical cystic fibrosis, certain neuropathologies including Alzheimer's disease linked to a mutation in the Tau protein, muscle atorphy which affects the SMN (survival of Motor Neuron) gene, depression linked to a serotonin splicing impairment, and certain cancers in which the global splicing process is affected (e.g. breast cancer, colon cancer and certain lymphomas), as well as viral diseases such as AIDS.
该发明涉及吲哚衍生物化合物及其用于制备药物的用途,该药物可用于治疗与细胞内预信使RNA剪接过程相关的疾病,如弗雷泽综合征、与染色体17相关的额颞叶痴呆症(一种帕金森病形式)、利综合征(一种脑病类型)、非典型囊性纤维化、某些神经病理学疾病,包括与Tau蛋白突变相关的阿尔茨海默病、影响SMN(运动神经元存活)基因的肌肉萎缩、与血清素剪接障碍相关的抑郁症,以及全局剪接过程受影响的某些癌症(例如乳腺癌、结肠癌和某些淋巴瘤),以及病毒性疾病,如艾滋病。