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全基因组测序在 500 个疑似遗传性疾病的亲子三体外发现的种系镶嵌现象:临床和遗传咨询意义。

Somatic mosaicism detected by genome-wide sequencing in 500 parent-child trios with suspected genetic disease: clinical and genetic counseling implications.

机构信息

Department of Medical Genetics, Faculty of Medicine, University of British Columbia, Vancouver, British Columbia, Canada V6H 3N1.

BC Children's Hospital Research Institute, Vancouver, British Columbia, Canada V5Z 4H4.

出版信息

Cold Spring Harb Mol Case Stud. 2021 Dec 9;7(6). doi: 10.1101/mcs.a006125. Print 2021 Dec.

Abstract

Identifying genetic mosaicism is important in establishing a diagnosis, assessing recurrence risk, and providing accurate genetic counseling. Next-generation sequencing has allowed for the identification of mosaicism at levels below those detectable by conventional Sanger sequencing or chromosomal microarray analysis. The CAUSES Clinic was a pediatric translational trio-based genome-wide (exome or genome) sequencing study of 500 families (531 children) with suspected genetic disease at BC Children's and Women's Hospitals. Here we present 12 cases of apparent mosaicism identified in the CAUSES cohort: nine cases of parental mosaicism for a disease-causing variant found in a child and three cases of mosaicism in the proband for a de novo variant. In six of these cases, there was no evidence of mosaicism on Sanger sequencing-the variant was not detected on Sanger sequencing in three cases, and it appeared to be heterozygous in three others. These cases are examples of six clinical manifestations of mosaicism: a proband with classical clinical features of mosaicism (e.g., segmental abnormalities of skin pigmentation or asymmetrical growth of bilateral body parts), a proband with unusually mild manifestations of a disease, a mosaic proband who is clinically indistinguishable from the constitutive phenotype, a mosaic parent with no clinical features of the disease, a mosaic parent with mild manifestations of the disease, and a family in which both parents are unaffected and two siblings have the same disease-causing constitutional mutation. Our data demonstrate the importance of considering the possibility of mosaicism whenever exome or genome sequencing is performed and that its detection via genome-wide sequencing can permit more accurate genetic counseling.

摘要

确定嵌合体对于建立诊断、评估复发风险和提供准确的遗传咨询非常重要。下一代测序技术使得能够在常规 Sanger 测序或染色体微阵列分析检测水平以下识别嵌合体。CAUSES 诊所是一个基于儿科转化的 trio 的全基因组(外显子或基因组)测序研究,涉及 BC 儿童医院的 500 个有疑似遗传疾病的家庭(531 个孩子)。在这里,我们介绍了在 CAUSES 队列中发现的 12 个明显嵌合体病例:9 个病例为父母的嵌合体,其子女携带致病变异;3 个病例为先证者携带新发变异的嵌合体。在这 6 个病例中,Sanger 测序均未发现嵌合体 - 在 3 个病例中未检测到 Sanger 测序中的变异,在另外 3 个病例中似乎为杂合子。这些病例是嵌合体的 6 种临床表现之一:先证者具有嵌合体的典型临床特征(例如,皮肤色素的节段性异常或双侧身体部分的不对称生长);先证者患有异常轻度疾病;临床表型与组成型表型无法区分的嵌合体先证者;无疾病临床特征的嵌合体父母;表现轻微疾病的嵌合体父母;以及父母双方均未受影响且有两个兄弟姐妹患有相同致病突变的家庭。我们的数据表明,无论进行外显子组还是基因组测序,都应考虑嵌合体的可能性,并且通过全基因组测序检测到的嵌合体可以进行更准确的遗传咨询。

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