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骨不全症伴正常胶原折叠中环孢素 B 的缺乏。

Lack of cyclophilin B in osteogenesis imperfecta with normal collagen folding.

机构信息

National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD 20892, USA.

出版信息

N Engl J Med. 2010 Feb 11;362(6):521-8. doi: 10.1056/NEJMoa0907705. Epub 2010 Jan 20.

Abstract

Osteogenesis imperfecta is a heritable disorder that causes bone fragility. Mutations in type I collagen result in autosomal dominant osteogenesis imperfecta, whereas mutations in either of two components of the collagen prolyl 3-hydroxylation complex (cartilage-associated protein [CRTAP] and prolyl 3-hydroxylase 1 [P3H1]) cause autosomal recessive osteogenesis imperfecta with rhizomelia (shortening of proximal segments of upper and lower limbs) and delayed collagen folding. We identified two siblings who had recessive osteogenesis imperfecta without rhizomelia. They had a homozygous start-codon mutation in the peptidyl-prolyl isomerase B gene (PPIB), which results in a lack of cyclophilin B (CyPB), the third component of the complex. The proband's collagen had normal collagen folding and normal prolyl 3-hydroxylation, suggesting that CyPB is not the exclusive peptidyl-prolyl cis-trans isomerase that catalyzes the rate-limiting step in collagen folding, as is currently thought.

摘要

成骨不全症是一种遗传性疾病,可导致骨骼脆弱。I 型胶原的突变导致常染色体显性成骨不全症,而胶原脯氨酰 3-羟化酶复合物的两个组成部分(软骨相关蛋白 [CRTAP] 和脯氨酰 3-羟化酶 1 [P3H1])的突变导致常染色体隐性成骨不全症伴骨干发育不良(四肢近段缩短)和胶原折叠延迟。我们鉴定了两名患有无骨干发育不良的隐性成骨不全症的同胞。他们在肽基脯氨酰异构酶 B 基因(PPIB)中存在纯合起始密码子突变,导致复合物的第三个组成部分亲环素 B(CyPB)缺失。先证者的胶原具有正常的胶原折叠和正常的脯氨酰 3-羟化,这表明 CyPB 不是目前认为的催化胶原折叠限速步骤的唯一肽基脯氨酰顺反异构酶。

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