Department of Pediatrics, Nagasaki University Graduate School of Biomedical Sciences, Nagasaki, Japan.
Department of Molecular Endocrinology, National Research Institute for Child Health and Development, Tokyo, Japan.
J Hum Genet. 2017 Oct;62(10):919-922. doi: 10.1038/jhg.2017.62. Epub 2017 Jun 8.
Silver-Russell syndrome (SRS) is a congenital developmental disorder characterized by intrauterine and postnatal growth failure, craniofacial features (including a triangular shaped face and broad forehead), relative macrocephaly, protruding forehead, body asymmetry and feeding difficulties. Hypomethylation of the H19 differentially methylated region (DMR) on chromosome 11p15.5 is the most common cause of the SRS phenotype. We report the first SRS patient with hypomethylation of the H19-DMR and maternally derived 15q11.2-q13.1 duplication. Although her clinical manifestations overlapped with those of previously reported SRS cases, the patient's intellectual disability and facial dysmorphic features were inconsistent with the SRS phenotype. Methylation analyses, array comparative genomic hybridization, and a FISH analysis revealed the hypomethylation of the H19-DMR and a maternally derived interstitial 5.7 Mb duplication at 15q11.2-q13.1 encompassing the Prader-Willi/Angelman critical region in the patient. On the basis of the genetic and clinical findings in the present and previously reported cases, it is unlikely that the 15q duplication in the patient led to the development of hypomethylation of the H19-DMR and it is reasonable to consider that the characteristic phenotype in the patient was caused by the coexistence of the two (epi)genetic conditions. Further studies are needed to clarify the mechanisms leading to methylation aberrations in SRS.
银-罗素综合征(SRS)是一种先天性发育障碍,其特征为宫内和产后生长发育迟缓、颅面特征(包括三角形脸和宽额头)、相对的大头畸形、前突的额头、身体不对称和喂养困难。11p15.5 上的 H19 差异甲基化区(DMR)的低甲基化是 SRS 表型的最常见原因。我们报告了首例 H19-DMR 低甲基化和母源性 15q11.2-q13.1 重复的 SRS 患者。尽管她的临床表现与先前报道的 SRS 病例重叠,但患者的智力障碍和面部畸形特征与 SRS 表型不一致。甲基化分析、阵列比较基因组杂交和 FISH 分析显示,患者的 H19-DMR 低甲基化和母源性 15q11.2-q13.1 内 5.7Mb 重复,该重复包含 Prader-Willi/Angelman 关键区域。基于本研究和先前报道病例的遗传和临床发现,患者的 15q 重复不太可能导致 H19-DMR 的低甲基化,因此可以合理地认为患者的特征表型是由这两种( epi )遗传条件共同导致的。需要进一步的研究来阐明导致 SRS 甲基化异常的机制。