Edwards M J, Wenstrup R J, Byers P H, Cohn D H
Ahmanson Department of Pediatrics, Steven Spielberg Pediatric Research Center, Cedars Sinai Medical Center, Los Angeles, California 90048.
Hum Mutat. 1992;1(1):47-54. doi: 10.1002/humu.1380010108.
We have determined that a man, ascertained because he fathered a child with lethal osteogenesis imperfecta (OI) with each of two partners, is mosaic in both his germline and somatic tissues for a mutation in the COL1A2 gene which encodes the pro alpha 2(I) chain of type I procollagen. His dermal fibroblasts were previously shown to synthesize a population of cysteine-containing alpha 2(I) chains that were posttranslationally overmodified. DNA sequence analysis of COL1A2 cDNAs demonstrated that the cysteine-containing chain resulted from a point mutation (G to T) in the first position of the codon for the glycine at residue 472 of the triple helical domain. Genomic DNA from the one available affected infant contained the mutant and normal COL1A2 alleles in equal proportion. Examination of DNA from several tissues of the father showed that the mutant allele was present in approximately 40% of his sperm, 80% of his lymphocytes, and nearly 100% of his dermal fibroblasts. Despite the high level of mosaicism detected in somatic tissues, the only phenotypic manifestation of OI in the proband was that he was shorter than his unaffected male relatives and had mild dentinogenesis imperfecta. Thermal stability of type I collagen molecules containing the substitution was decreased, but to a lesser extent than for a nonlethal cysteine for glycine substitution at residue 259 of alpha 2(I), indicating that this measure of molecular stability may be of limited use in explaining the pathogenesis of osteogenesis imperfecta.
我们已确定,有一名男子,因他与两名伴侣各育有一个患有致死性成骨不全症(OI)的孩子而被确诊,其生殖系和体细胞组织中编码I型前胶原原α2(I)链的COL1A2基因存在镶嵌现象。此前研究表明,他的皮肤成纤维细胞能合成一群翻译后过度修饰的含半胱氨酸的α2(I)链。对COL1A2 cDNA进行DNA序列分析显示,含半胱氨酸的链是由三螺旋结构域第472位甘氨酸密码子第一位的点突变(G突变为T)导致的。唯一一名患病婴儿的基因组DNA中,突变型和正常COL1A2等位基因比例相等。对该父亲多个组织的DNA检测表明,突变等位基因在其约40%的精子、80%的淋巴细胞以及几乎100%的皮肤成纤维细胞中存在。尽管在体细胞组织中检测到高度镶嵌现象,但先证者OI的唯一表型表现是他比未患病的男性亲属矮,且患有轻度牙本质发育不全。含有该替代突变的I型胶原分子热稳定性降低,但程度低于α2(I)链第259位非致死性半胱氨酸替代甘氨酸的情况,这表明这种分子稳定性指标在解释成骨不全症发病机制方面的作用可能有限。