Morava Eva, Zeevaert Renate, Korsch Eckhard, Huijben Karin, Wopereis Suzan, Matthijs Gert, Keymolen Kathelijn, Lefeber Dirk J, De Meirleir Linda, Wevers Ron A
Department of Pediatrics, Radboud University Nijmegen Medical Centre, 6500 HB Nijmegen, The Netherlands.
Eur J Hum Genet. 2007 Jun;15(6):638-45. doi: 10.1038/sj.ejhg.5201813. Epub 2007 Mar 14.
We describe the clinical and biochemical characteristics in three patients from two different families diagnosed with Congenital Disorder of Glycosylation type IIe owing to a defect in Conserved Oligomeric Golgi complex (COG)7; one of the eight subunits of the COG. The siblings and an unrelated single child of consanguineous parents presented with growth retardation, progressive, severe microcephaly, hypotonia, adducted thumbs, feeding problems by gastrointestinal pseudo-obstruction, failure to thrive, cardiac anomalies, wrinkled skin and episodes of extreme hyperthermia. A combined disorder in the biosynthesis of N- and O-linked glycosylation with hyposialylation was detected. Western blot analysis showed a severe reduction in the COG5 and 7 subunits of the COG. A homozygous, intronic splice site mutation (c.169+4A>C) of the COG7 gene was identified in all patients. The phenotype is similar to that previously described in two patients of North African ethnicity with the same mutation, except for the lack of skeletal anomalies and only a mild liver involvement in our patients. We suggest performing protein glycosylation studies and Western blot for the different COG subunits in patients with progressive microcephaly, growth retardation, hypotonia, adducted thumbs and cardiac defects, especially in association with skin anomalies or episodes of hyperthermia. The presence of the characteristic phenotype might warrant direct DNA analysis.
我们描述了来自两个不同家族的三名患者的临床和生化特征,这些患者因保守寡聚高尔基体复合物(COG)7(COG的八个亚基之一)存在缺陷而被诊断为IIe型先天性糖基化障碍。这些患者为近亲父母所生的兄弟姐妹及一名无血缘关系的独子,他们均表现出生长发育迟缓、进行性严重小头畸形、肌张力减退、拇指内收、因胃肠道假性梗阻导致的喂养问题、发育不良、心脏异常、皮肤起皱以及极度高热发作。检测发现N - 糖基化和O - 糖基化生物合成存在联合紊乱且唾液酸化不足。蛋白质免疫印迹分析显示COG的COG5和7亚基严重减少。在所有患者中均鉴定出COG7基因的纯合内含子剪接位点突变(c.169 + 4A>C)。除了我们的患者缺乏骨骼异常且仅有轻度肝脏受累外,该表型与先前描述的两名具有相同突变的北非裔患者相似。我们建议对患有进行性小头畸形、生长发育迟缓、肌张力减退、拇指内收和心脏缺陷的患者,尤其是伴有皮肤异常或高热发作的患者,进行蛋白质糖基化研究及针对不同COG亚基的蛋白质免疫印迹分析。特征性表型的存在可能需要进行直接的DNA分析。