Ito Shinya, Nagata Kazuhiro
Department of Molecular and Cellular Biology, Institute for Frontier Medical Sciences, Kyoto University, 53 Kawahara-cho, Sakyo-ku, Kyoto City, Kyoto 606-8397, Japan; Department of Molecular Biosciences, Faculty of Life Sciences, Kyoto Sangyo University, Motoyama, Kamigamo, Kita-ku, Kyoto 603-8555, Japan.
Department of Molecular Biosciences, Faculty of Life Sciences, Kyoto Sangyo University, Motoyama, Kamigamo, Kita-ku, Kyoto 603-8555, Japan; CREST, Japan Science and Technology Agency, Kyoto Sangyo University, Kyoto 603-8555, Japan.
Biochem Biophys Res Commun. 2016 Jan 15;469(3):437-42. doi: 10.1016/j.bbrc.2015.12.028. Epub 2015 Dec 12.
Osteogenesis imperfecta (OI) is a genetic disorder characterized by fragile bones. Most OI cases are caused by defects in type I collagen structure or synthesis. Mutations in collagen specific molecular chaperone Hsp47, specifically L78P and L326P, lead to OI, yet these mutants are not fully characterized. Here, we found that both Hsp47 mutants were structurally unstable and formed high molecular weight complexes. They were degraded by the ubiquitin-proteasome system, and the collagen-binding ability of the mutants was significantly lower than that of the wild type. Although the chemical chaperone 4-PBA partially restores the solubility of the Hsp47 OI mutants, collagen-binding activity of Hsp47 was not improved.
成骨不全症(OI)是一种以骨骼脆弱为特征的遗传性疾病。大多数OI病例是由I型胶原蛋白结构或合成缺陷引起的。胶原蛋白特异性分子伴侣Hsp47的突变,特别是L78P和L326P,会导致OI,但这些突变体尚未得到充分表征。在这里,我们发现这两种Hsp47突变体在结构上不稳定,并形成高分子量复合物。它们被泛素-蛋白酶体系统降解,并且突变体的胶原蛋白结合能力明显低于野生型。尽管化学伴侣4-PBA部分恢复了Hsp47 OI突变体的溶解度,但Hsp47的胶原蛋白结合活性并未得到改善。