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引用次数: 0
摘要
成骨不全症(OI)是一种异质性遗传性骨骼发育不良,其特征是骨脆性和畸形、生长缺陷以及其他继发性结缔组织缺陷。目前,OI 被认为是一种与胶原蛋白相关的疾病,是由于基因缺陷引起的,这些基因的蛋白产物与胶原蛋白相互作用,进行折叠、翻译后修饰、加工和运输,从而影响骨矿化和成骨细胞分化。这篇综述提供了有关 OI 遗传学的最新进展,包括显性和罕见 OI 形态的新进展,以及参与 OI 病理生理学的信号通路。我们特别强调了在 TENT5A、MESD、KDELR2 和 CCDC134 中发现的隐性突变,这些突变分别导致了第十九型、第二十型、第二十一型和第二十一型 OI。我们还详细回顾了将已知 OI 类型相互连接起来的新发现,这些发现可能是最终了解 OI 细胞和骨骼生物学中最终共同途径的基础。
Update on the Genetics of Osteogenesis Imperfecta.
Osteogenesis imperfecta (OI) is a heterogeneous heritable skeletal dysplasia characterized by bone fragility and deformity, growth deficiency, and other secondary connective tissue defects. OI is now understood as a collagen-related disorder caused by defects of genes whose protein products interact with collagen for folding, post-translational modification, processing and trafficking, affecting bone mineralization and osteoblast differentiation. This review provides the latest updates on genetics of OI, including new developments in both dominant and rare OI forms, as well as the signaling pathways involved in OI pathophysiology. There is a special emphasis on discoveries of recessive mutations in TENT5A, MESD, KDELR2 and CCDC134 whose causality of OI types XIX, XX, XXI and XXI, respectively, is now established and expends the complexity of mechanisms underlying OI to overlap LRP5/6 and MAPK/ERK pathways. We also review in detail new discoveries connecting the known OI types to each other, which may underlie an eventual understanding of a final common pathway in OI cellular and bone biology.
期刊介绍:
Calcified Tissue International and Musculoskeletal Research publishes original research and reviews concerning the structure and function of bone, and other musculoskeletal tissues in living organisms and clinical studies of musculoskeletal disease. It includes studies of cell biology, molecular biology, intracellular signalling, and physiology, as well as research into the hormones, cytokines and other mediators that influence the musculoskeletal system. The journal also publishes clinical studies of relevance to bone disease, mineral metabolism, muscle function, and musculoskeletal interactions.