Seeley Andrea H, Durham Mark A, Micale Mark A, Wesolowski Jeffrey, Foerster Bradley R, Martin Donna M
Department of Pediatrics and Communicable Diseases, University of Michigan Medical School, Ann Arbor, Michigan.
Am J Med Genet A. 2014 Aug;164A(8):2062-8. doi: 10.1002/ajmg.a.36569. Epub 2014 Apr 9.
Macrocerebellum is a rare condition characterized by enlargement of the cerebellum with conservation of the overall shape and cytoarchitecture. Here, we report on a child with a distinctive constellation of clinical features including macrocerebellum, epilepsy, apparent intellectual disability, dysautonomia, gut malrotation, and poor gut motility. Oligonucleotide chromosome microarray analysis identified a 16q24.1-q24.2 deletion that included four OMIM genes (FBXO31, MAP1LC3B, JPH3, and SLC7A5). Review of prior studies describing individuals with similar or overlapping16q24.1-q24.2 deletions identified no other reports of macrocerebellum. These observations highlight a potential genetic cause of this rare disorder and raise the possibility that one or more gene(s) in the 16q24.1-q24.2 interval regulate cerebellar development.
巨小脑是一种罕见病症,其特征为小脑增大而整体形状和细胞结构保持不变。在此,我们报告一名儿童,其具有一系列独特的临床特征,包括巨小脑、癫痫、明显的智力残疾、自主神经功能障碍、肠道旋转不良和肠道蠕动不佳。寡核苷酸染色体微阵列分析确定了一个16q24.1-q24.2缺失,其中包含四个OMIM基因(FBXO31、MAP1LC3B、JPH3和SLC7A5)。回顾先前描述具有相似或重叠16q24.1-q24.2缺失个体的研究,未发现其他关于巨小脑的报告。这些观察结果突出了这种罕见疾病的潜在遗传原因,并增加了16q24.1-q24.2区间内一个或多个基因调节小脑发育的可能性。