McGaughran Julie, Sinnott Stephen, Dastot-Le Moal Florence, Wilson Meredith, Mowat David, Sutton Bridget, Goossens Michel
Queensland Clinical Genetics Service, Royal Children's Hospital, Brisbane, Queensland, Australia.
Am J Med Genet A. 2005 Sep 1;137A(3):302-4. doi: 10.1002/ajmg.a.30896.
Mowat-Wilson syndrome (MWS) is a mental retardation syndrome associated with distinctive facial features, microcephaly, epilepsy, and a variable spectrum of congenital anomalies, including Hirschsprung disease (HSCR), agenesis of the corpus callosum, genitourinary abnormalities, and congenital heart disease. Heterozygous mutations or deletions involving the gene ZFHX1B (previously SIP1) [OMIM 605802] have recently been found to cause MWS. There have previously been no reports of a sibling recurrence of this syndrome. A brother and sister are described with clinical features of MWS, where both have the same truncating mutation in exon 8 of ZFHX1B. As their parents are phenotypically normal and do not have the mutation in lymphocyte-derived DNA, the most likely explanation is germ-line mosaicism.
莫瓦特-威尔逊综合征(MWS)是一种与特殊面部特征、小头畸形、癫痫以及一系列先天性异常相关的智力发育迟缓综合征,这些先天性异常包括先天性巨结肠(HSCR)、胼胝体发育不全、泌尿生殖系统异常和先天性心脏病。最近发现,涉及ZFHX1B基因(以前称为SIP1)[OMIM 605802]的杂合突变或缺失会导致MWS。此前尚无该综合征同胞复发的报道。本文描述了一对患有MWS临床特征的兄妹,他们在ZFHX1B基因第8外显子中具有相同的截短突变。由于他们的父母表型正常,且淋巴细胞衍生DNA中没有该突变,最可能的解释是生殖系嵌合现象。