Department of Cardiovascular Medicine, First Affiliated Hospital of the Medical College of Xi'an Jiaotong University, No. 277 Yanta West Road, Xi'an, 710061, Shaanxi, People's Republic of China.
Mol Cell Biochem. 2013 Oct;382(1-2):307-11. doi: 10.1007/s11010-013-1734-3. Epub 2013 Jun 22.
The LMNA gene, which encodes the nuclear envelope protein lamin A/C, is considered to be the most common autosomal disease gene associated with familial dilated cardiomyopathy. To date, each mutation of the LMNA gene has been associated with a specific disease phenotype. Clinical data, family histories, and blood samples were collected from 27 biological members of a family with dilated cardiomyopathy, prominently occurring as heart failure and conduction system disease with a high incidence of sudden cardiac death in young females. Twelve exons of the LMNA gene were screened for nucleotide alterations. A novel insertion mutation (nucleotide 1526insA, amino acid T510Y) in exon nine of the LMNA gene was identified in seven subjects (7/27, 25.9%). This reveals that the LMNA gene insertion mutation (T510Y frameshift mutation) can cause dilated cardiomyopathy, conduction system disease, and sudden cardiac death without skeletal myopathy, clinically manifested with early onset, severe symptoms, and poor prognosis.
编码核膜蛋白核纤层蛋白 A/C 的 LMNA 基因被认为是与家族性扩张型心肌病相关的最常见的常染色体疾病基因。迄今为止,LMNA 基因的每个突变都与特定的疾病表型相关。从一个扩张型心肌病的家族中收集了 27 位生物学成员的临床数据、家族史和血液样本,主要表现为心力衰竭和传导系统疾病,年轻女性的心脏性猝死发生率很高。对 LMNA 基因的 12 个外显子进行了核苷酸改变的筛查。在 9 号外显子中发现了一个新的插入突变(核苷酸 1526insA,氨基酸 T510Y),在 7 个受试者(7/27,25.9%)中发现了该突变。这表明 LMNA 基因插入突变(T510Y 移码突变)可导致扩张型心肌病、传导系统疾病和心脏性猝死,而无骨骼肌病,临床上表现为发病早、症状严重、预后差。