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着色性干皮病和科凯恩综合征:重叠的临床和生化表型。

Xeroderma pigmentosum and Cockayne syndrome: overlapping clinical and biochemical phenotypes.

作者信息

Greenhaw G A, Hebert A, Duke-Woodside M E, Butler I J, Hecht J T, Cleaver J E, Thomas G H, Horton W A

机构信息

Department of Pediatrics, University of Texas Medical School, Houston 77225.

出版信息

Am J Hum Genet. 1992 Apr;50(4):677-89.

Abstract

Two siblings are described whose clinical presentation of cutaneous photosensitivity and central nervous system dysfunction is strongly reminiscent of the DeSanctis-Cacchione syndrome (DCS) variant of xeroderma pigmentosum. An extensive clinical evaluation supported a diagnosis of DCS and documented previously unreported findings. In vitro fibroblast studies showed UV sensitivity that was two to three times that of normal controls. However, neither a post-UV-irradiation DNA excision-repair defect indicative of XP nor a semiconservative DNA replication defect indicative of XP variant was found. Rather, a failure of RNA synthesis to recover to normal levels after UV exposure was observed, a biochemical abnormality seen in Cockayne syndrome (CS), one of the premature-aging syndromes with clinical UV sensitivity. These patients, therefore, clinically have XP, but their biochemical characteristics suggest CS. The reason(s) for the severe neurologic disease, in light of the relatively mild cutaneous abnormalities, is unclear. Other cases with unusual fibroblast responses to irradiation have been noted in the literature and, along with the data from our patients, reinforce the notion of the complexity of DNA maintenance and repair.

摘要

本文描述了两名兄弟姐妹,他们的临床表现为皮肤光敏性和中枢神经系统功能障碍,这强烈让人联想到着色性干皮病的德桑蒂斯-卡乔内综合征(DCS)变体。广泛的临床评估支持了DCS的诊断,并记录了以前未报告的发现。体外成纤维细胞研究显示紫外线敏感性是正常对照的两到三倍。然而,既未发现表明着色性干皮病的紫外线照射后DNA切除修复缺陷,也未发现表明着色性干皮病变体的半保留DNA复制缺陷。相反,观察到紫外线暴露后RNA合成未能恢复到正常水平,这是科凯恩综合征(CS)中出现的一种生化异常,CS是一种具有临床紫外线敏感性的早衰综合征。因此,这些患者临床上患有着色性干皮病,但其生化特征提示为科凯恩综合征。鉴于相对较轻的皮肤异常,严重神经疾病的原因尚不清楚。文献中还提到了其他成纤维细胞对辐射有异常反应的病例,连同我们患者的数据,强化了DNA维持和修复复杂性的概念。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7370/1682634/0dd25e30c5c6/ajhg00075-0022-a.jpg

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