Suter Bernhard, Treadwell-Deering Diane, Zoghbi Huda Y, Glaze Daniel G, Neul Jeffrey L
Section of Child Neurology and Developmental Neuroscience, Department of Pediatrics, Baylor College of Medicine, Houston, TX, USA.
J Autism Dev Disord. 2014 Mar;44(3):703-11. doi: 10.1007/s10803-013-1902-z.
Mutations in Methyl-CpG-Binding protein 2 (MECP2) are commonly associated with the neurodevelopmental disorder Rett syndrome (RTT). However, some people with RTT do not have mutations in MECP2, and interestingly there have been people identified with MECP2 mutations that do not have the clinical features of RTT. In this report we present four people with neurodevelopmental abnormalities and clear RTT-disease causing MECP2 mutation but lacking the characteristic clinical features of RTT. One patient's symptoms suggest an extension of the known spectrum of MECP2 associated phenotypes to include global developmental delay with obsessive compulsive disorder and attention deficit hyperactivity disorder. These results reemphasize that RTT should remain a clinical diagnosis, based on the recent consensus criteria.
甲基-CpG-结合蛋白2(MECP2)突变通常与神经发育障碍雷特综合征(RTT)相关。然而,一些患有RTT的人并没有MECP2突变,有趣的是,也有被鉴定出具有MECP2突变但没有RTT临床特征的人。在本报告中,我们介绍了四名有神经发育异常且有明确的导致RTT疾病的MECP2突变但缺乏RTT特征性临床特征的人。一名患者的症状表明,已知的MECP2相关表型谱有所扩展,包括伴有强迫症和注意力缺陷多动障碍的全面发育迟缓。这些结果再次强调,根据最近的共识标准,RTT应仍然是一种临床诊断。