Department of Neurology, Hospital Universitario 12 de Octubre, Madrid, Spain.
Group of Neurodegenerative Diseases, Instituto de Investigación Hospital 12 de Octubre (I+12), Madrid, Spain.
J Alzheimers Dis. 2021;84(1):73-78. doi: 10.3233/JAD-210648.
The haploinsufficiency of the methyl-binding domain protein 5 (MBD5) gene has been identified as the determinant cause of the neuropsychiatric disorders grouped under the name MBD5-neurodevelopment disorders (MAND). MAND includes patients with intellectual disability, behavioral problems, and seizures with a static clinical course. However, a few reports have suggested regression. We describe a non-intellectually disabled female, with previous epilepsy and personality disorder, who developed early-onset dementia. The extensive etiologic study revealed a heterozygous nonsense de novo pathogenic variant in the MBD5 gene. This finding could support including the MBD5 gene in the study of patients with atypical early-onset dementia.
甲基结合域蛋白 5(MBD5)基因的单倍体不足已被确定为神经精神障碍的决定因素,这些障碍被归为 MBD5-神经发育障碍(MAND)。MAND 包括伴有智力残疾、行为问题和癫痫发作且临床病程呈静态的患者。然而,有少数报道提示病情有进展。我们描述了一位非智力残疾的女性,她之前患有癫痫和人格障碍,后发展为早发性痴呆。广泛的病因学研究发现 MBD5 基因存在杂合无义新生致病性变异。这一发现可能支持将 MBD5 基因纳入非典型早发性痴呆患者的研究。