Laboratory of Medical Genetics, IRCCS Casa Sollievo Della Sofferenza Hospital, San Giovanni Rotondo, Italy.
Hum Genet. 2010 Jul;128(1):3-26. doi: 10.1007/s00439-010-0827-2. Epub 2010 May 1.
Copy number variants (CNVs) of the Williams-Beuren syndrome (WBS) 7q11.23 region are responsible for neurodevelopmental disorders with multi-system involvement and variable expressivity. Typical features of WBS microdeletion comprise a recognizable pattern of facial dysmorphisms, supravalvular aortic stenosis, connective tissue abnormalities, hypercalcemia, and a distinctive neurobehavioral phenotype. Conversely, the phenotype of patients carrying the 7q11.23 reciprocal duplications includes less distinctive facial dysmorphisms and prominent speech delay. The common deletion/duplication ranges in size from 1.5 to 1.8 Mb and encompasses approximately 28 genes. This region is flanked by low copy repeats (LCRs) with greater than ~97% identity, which can mediate non-allelic homologous recombination resulting from misalignment of LCRs during meiosis. A clear genotype-phenotype correlation has been established in WBS only for the elastin gene, which is responsible for the vascular and connective tissue abnormalities. The molecular substrates underlying the other clinical features of 7q11.23 CNVs, including the neurocognitive phenotypes, are still debated. Recent studies suggest that besides the role of the genes in the deleted/duplicated interval, multiple factors such as regulatory sequences, epigenetic mechanisms, parental origin of the CNV, and nucleotide variations in the non-deleted/duplicated allele may be important in determining the variable expressivity of 7q11.23 CNV phenotypes. Here, we review the clinical and molecular findings and the recent insights on genomic disorders associated with CNVs involving the 7q11.23 region.
威廉姆斯-比伦综合征(WBS)7q11.23 区域的拷贝数变异(CNVs)可导致多系统受累和表现度可变的神经发育障碍。WBS 微缺失的典型特征包括可识别的面部畸形、主动脉瓣上狭窄、结缔组织异常、高钙血症和独特的神经行为表型。相反,携带 7q11.23 反向重复的患者的表型包括不太明显的面部畸形和明显的言语延迟。常见的缺失/重复大小为 1.5 至 1.8 Mb,包含约 28 个基因。该区域由低拷贝重复序列(LCRs)侧翼包围,具有大于约 97%的同源性,这些 LCRs 在减数分裂过程中可能会因 LCR 错位而发生非等位基因同源重组。WBS 中仅建立了明确的基因型-表型相关性,弹性蛋白基因是导致血管和结缔组织异常的原因。7q11.23 CNVs 其他临床特征的分子基础,包括神经认知表型,仍存在争议。最近的研究表明,除了缺失/重复间隔内的基因作用外,多个因素如调节序列、表观遗传机制、CNV 的亲本来源以及非缺失/重复等位基因中的核苷酸变异,可能在决定 7q11.23 CNV 表型的可变表达中起着重要作用。在这里,我们综述了与涉及 7q11.23 区域的 CNVs 相关的基因组疾病的临床和分子发现以及最近的见解。