Compositions and method for stabilizing transthyretin and inhibiting transthyretin misfolding
申请人:The Scripps Research Institute
公开号:EP1988397A1
公开(公告)日:2008-11-05
Kinetic stabilization of the native state of transthyretin is an effective mechanism for preventing protein misfolding. Because transthyretin misfolding plays an important role in transthyretin amyloid diseases, inhibiting such misfolding can be used as an effective treatment or prophylaxis for such diseases. Treatment methods, screening methods, as well as specific transthyretin stabilizing compounds are disclosed.
转甲状腺素原生态的动力学稳定是防止蛋白质错误折叠的有效机制。由于转甲状腺素错误折叠在转甲状腺素淀粉样变性疾病中起着重要作用,因此抑制这种错误折叠可作为治疗或预防此类疾病的有效方法。本文公开了治疗方法、筛选方法以及特异性转甲状腺素稳定化合物。