Richards F M, Crossey P A, Phipps M E, Foster K, Latif F, Evans G, Sampson J, Lerman M I, Zbar B, Affara N A
Cambridge University Department of Pathology, UK.
Hum Mol Genet. 1994 Apr;3(4):595-8. doi: 10.1093/hmg/3.4.595.
Von Hippel-Lindau disease is a dominantly inherited familial cancer syndrome characterised by the development of retinal angiomatosis, cerebellar and spinal hemangioblastoma, renal cell carcinoma, phaeochromocytoma and pancreatic tumours. A cDNA (g7) which detects frequent genomic rearrangements in VHL disease patients on Southern analysis, and contains the partial coding sequence of the VHL gene has been isolated recently. To characterise the nature of the genomic rearrangements in VHL disease we initially screened 116 patients with VHL disease and identified 22 patients (19%) with abnormal fragments in EcoR1 digested DNA probes with g7. We then established that the coding sequence contained within g7 is represented in 3 exons, and design exon specific probes to investigate the 22 patients with genomic rearrangements. All 22 patients were demonstrated to have germline deletions, but the deletions were heterogeneous with 7 patients having deletions confined to the 5' exon 1, and 8 with nonoverlapping deletions of exon 3. In 7 unrelated patients, including 2 new mutations, the germline deletions were similar in size and position. There was no relationship between the clinical phenotype and the deletion of individual exons. Although phaeochromocytoma was less frequent in kindreds with germline deletions than those without detectable deletions, the difference was not statistically significant (1/19 versus 16/72 respectively, chi 2 = 1.84 p > 0.1).
冯·希佩尔-林道病是一种常染色体显性遗传的家族性癌症综合征,其特征为视网膜血管瘤、小脑和脊髓血管母细胞瘤、肾细胞癌、嗜铬细胞瘤及胰腺肿瘤的发生。最近已分离出一种互补DNA(g7),经Southern分析,它能检测出冯·希佩尔-林道病患者中常见的基因组重排,并且包含VHL基因的部分编码序列。为了明确冯·希佩尔-林道病中基因组重排的性质,我们首先筛查了116例冯·希佩尔-林道病患者,并用g7在经EcoR1消化的DNA探针中鉴定出22例(19%)有异常片段的患者。然后我们确定g7内包含的编码序列由3个外显子表示,并设计外显子特异性探针来研究这22例有基因组重排的患者。所有22例患者均被证明存在种系缺失,但缺失情况是异质性的,7例患者的缺失局限于5'外显子1,8例患者的外显子3有不重叠缺失。在7例无亲缘关系的患者中,包括2例新突变,种系缺失在大小和位置上相似。临床表型与单个外显子的缺失之间没有关系。虽然与无种系缺失的家族相比,种系缺失家族中的嗜铬细胞瘤发病率较低,但差异无统计学意义(分别为1/19和16/72,卡方检验=1.84,p>0.1)。