Heilstedt Heidi A, Ballif Blake C, Howard Leslie A, Lewis Richard A, Stal Samuel, Kashork Catherine D, Bacino Carlos A, Shapira Stuart K, Shaffer Lisa G
Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.
Am J Hum Genet. 2003 May;72(5):1200-12. doi: 10.1086/375179. Epub 2003 Apr 8.
Monosomy 1p36 is the most common terminal deletion syndrome. This contiguous gene deletion syndrome is presumably caused by haploinsufficiency of a number of genes. We have constructed a contig of overlapping large-insert clones for the most distal 10.5 Mb of 1p36, evaluated the deletion sizes in 61 subjects with monosomy 1p36 from 60 families, and created a natural deletion panel. We found pure terminal deletions, interstitial deletions, derivative chromosomes, and more complex rearrangements. Breakpoints were "binned" into 0.5-Mb regions. Analyses revealed some clustering of breakpoints but no single common breakpoint. Determination of the parental origin showed that 60% of de novo 1p36 terminal deletions arose from the maternally inherited chromosome. Of the 61 subjects, 30 were examined systematically through a protocol at the Texas Children's Hospital General Clinical Research Center. Specifically, we report hearing evaluations, palatal and ophthalmological examinations, echocardiograms, neurological assessments, and thyroid function tests. To our knowledge, this systematic molecular and clinical characterization of monosomy 1p36 is the largest and most comprehensive study of this deletion syndrome to date. Many cytogenetically visible, apparent terminal deletions are more complex than anticipated by cytogenetics, as revealed at the molecular level by our study. Our clinical findings allow for the more accurate recognition of the syndrome and for proper medical evaluation.
1p36单体是最常见的末端缺失综合征。这种相邻基因缺失综合征可能是由多个基因的单倍剂量不足引起的。我们构建了一个重叠大片段克隆的重叠群,覆盖1p36最远端的10.5 Mb区域,评估了来自60个家庭的61例1p36单体患者的缺失大小,并创建了一个自然缺失面板。我们发现了纯末端缺失、中间缺失、衍生染色体以及更复杂的重排。断点被“分类”到0.5 Mb的区域。分析显示断点有一些聚集,但没有单一的共同断点。亲代来源的确定表明,60%的新发1p36末端缺失来自母系遗传的染色体。在这61例患者中,30例在德克萨斯儿童医院综合临床研究中心通过一项方案进行了系统检查。具体而言,我们报告了听力评估、腭部和眼科检查、超声心动图、神经学评估以及甲状腺功能测试。据我们所知,这项对1p36单体的系统分子和临床特征分析是迄今为止对这种缺失综合征规模最大、最全面的研究。正如我们的研究所揭示的,在分子水平上,许多细胞遗传学上可见的明显末端缺失比细胞遗传学预期的更为复杂。我们的临床发现有助于更准确地识别该综合征并进行适当的医学评估。