长型 Plod2 的缺失会导致布吕克综合征--成骨不全症--的挛缩。

IF 5.1 1区 医学 Q1 ENDOCRINOLOGY & METABOLISM Journal of Bone and Mineral Research Pub Date : 2024-09-02 DOI:10.1093/jbmr/zjae124
Alexander Kot, Cora Chun, Jorge H Martin, Davis Wachtell, David Hudson, MaryAnn Weis, Haley Marks, Siddharth Srivastava, David R Eyre, Ivan Duran, Jennifer Zieba, Deborah Krakow
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引用次数: 0

摘要

布吕克综合征(Bruck Syndrome)是一种常染色体隐性遗传的成骨不全症(OI),由 PLOD2 或 FKBP10 的双拷贝变异引起,以关节挛缩、骨脆性、身材矮小和脊柱侧弯为特征。PLOD2 编码 LH2,它能羟化 I 型胶原端肽赖氨酸,这是胶原交联的关键步骤。Plod2 基因全基因敲除小鼠模型受限于早期胚胎致死率,因此人们对 PLOD2 在骨骼发生过程中的作用还不甚了解。我们生成了一个新型 Plod2 小鼠品系,该品系是在两个无关的布吕克综合征患者中发现的一个变体:PLOD2 c.1559dupC,预示着一个框架移位和长异构体 LH2b 的缺失。在小鼠中,这种重复导致 LH2b mRNA 的缺失以及 LH2 蛋白总量的显著减少。该模型(Plod2fs/fs)一直存活到 E18.5,但其基因型频率并非孟德尔型。同基因缺失模型再现了布鲁克综合征中的关节挛缩,并显示骨骼中缺乏 I 型胶原端肽赖氨酸羟基化。用Scleraxis-GFP对Plod2fs/fs小鼠的肌腱进行基因标记后发现,到E18.5时,小鼠前肢的伸肌腱缺失,发育研究显示,伸肌腱在E14.5时已经发育,但在E16.5时开始缺失。二次谐波生成显示肌腱 I 型胶原纤维组织异常,表明肌腱结构异常。通过μCT和拉曼光谱对骨骼进行的表征显示骨矿化水平正常。这项工作强调了I型胶原蛋白在肌腱和骨骼中正常交联的重要性,为我们进一步了解布吕克综合征提供了一个很有前景的新小鼠模型。
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Loss of the long form of Plod2 phenocopies contractures of Bruck syndrome-osteogenesis imperfecta.

Bruck syndrome is an autosomal recessive form of osteogenesis imperfecta caused by biallelic variants in PLOD2 or FKBP10 and is characterized by joint contractures, bone fragility, short stature, and scoliosis. PLOD2 encodes LH2, which hydroxylates type I collagen telopeptide lysines, a critical step for collagen crosslinking. The Plod2 global knockout mouse model is limited by early embryonic lethality, and thus, the role of PLOD2 in skeletogenesis is not well understood. We generated a novel Plod2 mouse line modeling a variant identified in two unrelated individuals with Bruck syndrome: PLOD2 c.1559dupC, predicting a frameshift and loss of the long isoform LH2b. In the mouse, the duplication led to loss of LH2b mRNA as well as significantly reduced total LH2 protein. This model, Plod2fs/fs, survived up to E18.5 although in non-Mendelian genotype frequencies. The homozygous frameshift model recapitulated the joint contractures seen in Bruck syndrome and had indications of absent type I collagen telopeptide lysine hydroxylation in bone. Genetically labeling tendons with Scleraxis-GFP in Plod2fs/fs mice revealed the loss of extensor tendons in the forelimb by E18.5, and developmental studies showed extensor tendons developed through E14.5 but were absent starting at E16.5. Second harmonic generation showed abnormal tendon type I collagen fiber organization, suggesting structurally abnormal tendons. Characterization of the skeleton by μCT and Raman spectroscopy showed normal bone mineralization levels. This work highlights the importance of properly crosslinked type I collagen in tendon and bone, providing a promising new mouse model to further our understanding of Bruck syndrome.

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来源期刊
Journal of Bone and Mineral Research
Journal of Bone and Mineral Research 医学-内分泌学与代谢
CiteScore
11.30
自引率
6.50%
发文量
257
审稿时长
2 months
期刊介绍: The Journal of Bone and Mineral Research (JBMR) publishes highly impactful original manuscripts, reviews, and special articles on basic, translational and clinical investigations relevant to the musculoskeletal system and mineral metabolism. Specifically, the journal is interested in original research on the biology and physiology of skeletal tissues, interdisciplinary research spanning the musculoskeletal and other systems, including but not limited to immunology, hematology, energy metabolism, cancer biology, and neurology, and systems biology topics using large scale “-omics” approaches. The journal welcomes clinical research on the pathophysiology, treatment and prevention of osteoporosis and fractures, as well as sarcopenia, disorders of bone and mineral metabolism, and rare or genetically determined bone diseases.
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