Orthopaedic Surgery, David Geffen School of Medicine at University of California at Los Angeles, Los Angeles, CA 90095, United States.
Human Genetics, David Geffen School of Medicine at University of California at Los Angeles, Los Angeles, CA 90095, United States.
J Bone Miner Res. 2024 Sep 2;39(9):1240-1252. doi: 10.1093/jbmr/zjae124.
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.
Bruck 综合征是一种常染色体隐性形式的成骨不全症,由 PLOD2 或 FKBP10 的双等位基因变异引起,其特征为关节挛缩、骨骼脆弱、身材矮小和脊柱侧凸。PLOD2 编码 LH2,该蛋白羟化 I 型胶原末端赖氨酸,这是胶原交联的关键步骤。Plod2 全局敲除小鼠模型由于早期胚胎致死而受到限制,因此,PLOD2 在骨骼发生中的作用尚未得到很好的理解。我们生成了一种新型 Plod2 小鼠模型,该模型模拟了在两个无关的 Bruck 综合征患者中发现的变体:PLOD2 c.1559dupC,预测移码并导致长型 LH2b 丢失。在小鼠中,该重复导致 LH2b mRNA 丢失以及总 LH2 蛋白显著减少。尽管在非孟德尔基因型频率下,这种模型(Plod2fs/fs)可存活至 E18.5。该纯合移码模型重现了 Bruck 综合征所见的关节挛缩,并表明骨骼中 I 型胶原末端赖氨酸羟化缺失。在 Plod2fs/fs 小鼠中用 Scleraxis-GFP 遗传标记肌腱显示 E18.5 时前肢伸肌腱丢失,发育研究表明伸肌腱在 E14.5 时发育,但从 E16.5 开始缺失。二次谐波产生显示异常的肌腱 I 型胶原纤维组织,表明肌腱结构异常。μCT 和拉曼光谱学对骨骼的特征分析表明骨矿物质化水平正常。这项工作强调了正确交联的 I 型胶原在肌腱和骨骼中的重要性,为进一步了解 Bruck 综合征提供了有前景的新型小鼠模型。