Department of Psychiatry, David Geffen School of Medicine, University of California Los Angeles, 695 Charles E. Young Drive South, #3506C Gonda Neuroscience and Genetics Research Center, Los Angeles, CA, 90095, USA.
Department of Neurosciences and Reproductive and Odontostomatological Sciences, Federico II University, Naples, Italy.
Neurogenetics. 2019 May;20(2):99-102. doi: 10.1007/s10048-019-00571-8. Epub 2019 Mar 21.
Primary familial brain calcification (PFBC) is a rare disorder mostly characterized by calcium deposits in the basal ganglia and a wide spectrum of neurologic and psychiatric symptoms, typically inherited as an autosomal dominant trait. Recently, MYORG was reported as the first autosomal recessive causal gene in PFBC patients of Chinese and Middle Eastern origin. Herein, we describe the first PFBC patient of European descent found to carry a novel homozygous MYORG mutation (p.N511Tfs*243). Interestingly, the patient's father, a heterozygous carrier of the same mutation, showed diffuse bilateral cerebral calcifications with no symptoms other than very mild postural tremor.
原发性家族性脑钙化为一种罕见疾病,主要表现为基底节区钙沉积和广泛的神经和精神症状,通常呈常染色体显性遗传。最近,MYORG 被报道为中国和中东地区原发性家族性脑钙化患者的首个常染色体隐性致病基因。本研究中,我们描述了首例发现携带新型纯合 MYORG 突变(p.N511Tfs*243)的欧洲血统原发性家族性脑钙化患者。有趣的是,该患者的父亲为同一突变的杂合子携带者,表现为弥漫性双侧大脑钙化,除姿势性震颤非常轻微外无其他症状。