Department of Biosciences and Nutrition, Center for Biosciences, Karolinska Institutet, Huddinge SE-14183, Sweden.
J Biol Chem. 2012 Sep 28;287(40):33512-22. doi: 10.1074/jbc.M112.366450. Epub 2012 Aug 14.
Hutchinson-Gilford progeria syndrome (HGPS) is a very rare genetic disorder that is characterized by multiple features of premature aging and largely affects tissues of mesenchymal origin. In this study, we describe the development of a tissue-specific mouse model that overexpresses the most common HGPS mutation (LMNA, c.1824C>T, p.G608G) in osteoblasts. Already at the age of 5 weeks, HGPS mutant mice show growth retardation, imbalanced gait and spontaneous fractures. Histopathological examination revealed an irregular bone structure, characterized by widespread loss of osteocytes, defects in mineralization, and a hypocellular red bone marrow. Computerized tomography analysis demonstrated impaired skeletal geometry and altered bone structure. The skeletal defects, which resemble the clinical features reported for bone disease in HGPS patients, was associated with an abnormal osteoblast differentiation. The osteoblast-specific expression of the HGPS mutation increased DNA damage and affected Wnt signaling. In the teeth, irregular dentin formation, as was previously demonstrated in human progeria cases, caused severe dental abnormalities affecting the incisors. The observed phenotype also shows similarities to reported bone abnormalities in aging mice and may therefore help to uncover general principles of the aging process.
亨廷顿舞蹈症-吉福德早衰综合征(Hutchinson-Gilford progeria syndrome,HGPS)是一种非常罕见的遗传性疾病,其特征是多种过早衰老的特征,并且主要影响间充质来源的组织。在这项研究中,我们描述了一种组织特异性的小鼠模型的开发,该模型在成骨细胞中过表达最常见的 HGPS 突变(LMNA,c.1824C>T,p.G608G)。HGPS 突变小鼠早在 5 周龄时就表现出生长迟缓、步态不平衡和自发性骨折。组织病理学检查显示骨结构不规则,表现为广泛的破骨细胞丢失、矿化缺陷和细胞减少的红骨髓。计算机断层扫描分析显示骨骼几何形状受损和骨结构改变。这些骨骼缺陷类似于 HGPS 患者骨骼疾病的临床特征,与异常的成骨细胞分化有关。HGPS 突变的成骨细胞特异性表达增加了 DNA 损伤,并影响了 Wnt 信号通路。在牙齿中,不规则的牙本质形成,如以前在人类早衰病例中所证明的,导致严重的牙齿异常,影响切牙。观察到的表型也与报道的衰老小鼠的骨骼异常相似,因此可能有助于揭示衰老过程的一般原则。