Genetic and Metabolic Central Laboratory, Guangxi Maternal and Child Health Hospital, Nanning, Guangxi 530023, P.R. China.
Nanning Region Center for Disease Prevention and Control, Nanning, Guangxi 530023, P.R. China.
Mol Med Rep. 2018 Mar;17(3):4433-4439. doi: 10.3892/mmr.2018.8436. Epub 2018 Jan 16.
Osteogenesis imperfecta (OI) is a rare congenital disorder characterized by bone fragility and fractures, and associated with bone deformity, short stature, dentin, ligament and blue‑gray eye sclera. OI is caused by a heterozygous mutation in collagen α‑1(I) chain (COL1A1) or collagen α‑2(I) chain (COL1A2) genes that encode α chains of type I collagen. Collagen α chain peptide contains an N‑propeptide, which has a role in assembly and processing of collagen. Point mutations in the N‑propeptide domain appear to trigger OI. In the present study, a novel heterozygous missense mutation, c.281T>A (p.Val94Asp), was identified in the von Willebrand C domain of N‑terminal of type I collagen in an individual with type IV OI. The majority of N‑terminal mutations are associated with OI/Ehlers‑Danlos syndrome (EDS); however, in the present study, the affected individual did not suffer from EDS and the level of serum phosphorus of the patient was low (0.67 mmol/l). A number of clinical phenotypes were observed at the same variation site or in the same region on the polypeptide chain of COL1A, which suggests that additional genetic and environmental factors may influence the severity of OI. The present study may provide insight into the phenotype‑genotype association in collagen-associated diseases and improve clinical diagnosis of OI.
成骨不全症(OI)是一种罕见的先天性疾病,其特征为骨骼脆弱和骨折,并且与骨骼畸形、身材矮小、牙本质、韧带和蓝灰色巩膜有关。OI 是由胶原蛋白 α-1(I)链(COL1A1)或胶原蛋白 α-2(I)链(COL1A2)基因的杂合突变引起的,这些基因编码 I 型胶原蛋白的 α 链。胶原蛋白 α 链肽含有 N-前肽,其在胶原蛋白的组装和加工中起作用。N-前肽结构域中的点突变似乎会引发 OI。在本研究中,在患有 IV 型 OI 的个体的 I 型胶原蛋白 N 端的 von Willebrand C 结构域中鉴定出一种新型杂合错义突变,c.281T>A(p.Val94Asp)。大多数 N-端突变与 OI/Ehlers-Danlos 综合征(EDS)相关;然而,在本研究中,受影响的个体没有患 EDS,并且患者的血清磷水平较低(0.67mmol/l)。在 COL1A 的同一变异位点或多肽链的同一区域观察到多种临床表型,这表明其他遗传和环境因素可能会影响 OI 的严重程度。本研究可能为胶原蛋白相关疾病的表型-基因型关联提供新的见解,并改善 OI 的临床诊断。