Yokoyama Takeshi, Mizuguchi Mineyuki
Graduate School of Medicine and Pharmaceutical Sciences, University of Toyama.
Graduate School of Innovative Life Science, University of Toyama.
Biol Pharm Bull. 2018;41(7):979-984. doi: 10.1248/bpb.b18-00166.
Hereditary transthyretin (TTR)-related amyloidosis is caused by mutations in the TTR gene. The mutations destabilize the tetramer and/or monomer of TTR, and thus the stabilization of TTR is a key strategy for the treatment of TTR-related amyloidosis. In this review, we summarized the natural products and synthetic compounds that have been shown to inhibit the amyloidogenesis of TTR. The stabilizers and/or the amyloid fibril disrupters isolated from natural sources may become lead compounds for the treatment of TTR-related amyloidosis.
遗传性转甲状腺素蛋白(TTR)相关淀粉样变性由TTR基因突变引起。这些突变会使TTR的四聚体和/或单体不稳定,因此稳定TTR是治疗TTR相关淀粉样变性的关键策略。在本综述中,我们总结了已被证明可抑制TTR淀粉样变的天然产物和合成化合物。从天然来源分离出的稳定剂和/或淀粉样纤维破坏剂可能成为治疗TTR相关淀粉样变性的先导化合物。