Mefford Heather C, Clauin Severine, Sharp Andrew J, Moller Rikke S, Ullmann Reinhard, Kapur Raj, Pinkel Dan, Cooper Gregory M, Ventura Mario, Ropers H Hilger, Tommerup Niels, Eichler Evan E, Bellanne-Chantelot Christine
Department of Genome Sciences, University of Washington, Seattle, WA 98195, USA.
Am J Hum Genet. 2007 Nov;81(5):1057-69. doi: 10.1086/522591. Epub 2007 Sep 26.
Most studies of genomic disorders have focused on patients with cognitive disability and/or peripheral nervous system defects. In an effort to broaden the phenotypic spectrum of this disease model, we assessed 155 autopsy samples from fetuses with well-defined developmental pathologies in regions predisposed to recurrent rearrangement, by array-based comparative genomic hybridization. We found that 6% of fetal material showed evidence of microdeletion or microduplication, including three independent events that likely resulted from unequal crossing-over between segmental duplications. One of the microdeletions, identified in a fetus with multicystic dysplastic kidneys, encompasses the TCF2 gene on 17q12, previously shown to be mutated in maturity-onset diabetes, as well as in a subset of pediatric renal abnormalities. Fine-scale mapping of the breakpoints in different patient cohorts revealed a recurrent 1.5-Mb de novo deletion in individuals with phenotypes that ranged from congenital renal abnormalities to maturity-onset diabetes of the young type 5. We also identified the reciprocal duplication, which appears to be enriched in samples from patients with epilepsy. We describe the first example of a recurrent genomic disorder associated with diabetes.
大多数基因组疾病研究都集中在患有认知障碍和/或外周神经系统缺陷的患者身上。为了拓宽这种疾病模型的表型谱,我们通过基于阵列的比较基因组杂交技术,评估了155份来自易发生反复重排区域、具有明确发育病理学特征的胎儿尸检样本。我们发现6%的胎儿样本显示有微缺失或微重复的证据,其中包括三个可能由节段性重复之间不等交换导致的独立事件。在一个患有多囊性发育不良肾的胎儿中鉴定出的一个微缺失,包含17q12上的TCF2基因,该基因先前已被证明在成年发病型糖尿病以及一部分儿科肾脏异常中发生突变。对不同患者队列中断点的精细定位揭示了在表型从先天性肾脏异常到青年型5成年发病型糖尿病不等的个体中存在一个反复出现的1.5兆碱基的新生缺失。我们还鉴定出了相互重复,其似乎在癫痫患者的样本中富集。我们描述了与糖尿病相关的反复出现的基因组疾病的首个实例。
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