Hegele Robert A, Cao Henian, Liu Dora M, Costain Gary A, Charlton-Menys Valentine, Rodger N Wilson, Durrington Paul N
Robarts Research Institute, 406-100 Perth Drive, London, Ontario, Canada N6A 5K8.
Am J Hum Genet. 2006 Aug;79(2):383-9. doi: 10.1086/505885. Epub 2006 Jun 5.
The etiology of acquired partial lipodystrophy (APL, also called "Barraquer-Simons syndrome") is unknown. Genomic DNA mutations affecting the nuclear lamina protein lamin A cause inherited partial lipodystrophy but are not found in patients with APL. Because it also encodes a nuclear lamina protein (lamin B2) and its genomic structure was recently reannotated, we sequenced LMNB2 as a candidate gene in nine white patients with APL. In four patients, we found three new rare mutations in LMNB2: intron 1 -6G-->T, exon 5 c.643G-->A (p.R215Q; in two patients), and exon 8 c.1218G-->A (p.A407T). The combined frequency of these mutations was 0.222 in the patients with APL, compared with 0.0018 in a multiethnic control sample of 1,100 subjects (P = 2.1 x 10-7) and 0.0045 in a sample of 330 white controls (P = 1.2 x 10-5). These novel heterozygous mutations are the first reported for LMNB2, are the first reported among patients with APL, and indicate how sequencing of a reannotated candidate gene can reveal new disease-associated mutations.
获得性部分脂肪营养不良(APL,也称为“巴拉奎尔 - 西蒙斯综合征”)的病因尚不清楚。影响核纤层蛋白A型的基因组DNA突变会导致遗传性部分脂肪营养不良,但在APL患者中未发现此类突变。由于它还编码一种核纤层蛋白(核纤层蛋白B2)且其基因组结构最近重新注释,我们对9名患有APL的白人患者的LMNB2基因作为候选基因进行了测序。在4名患者中,我们在LMNB2中发现了3个新的罕见突变:内含子1 -6G→T、外显子5 c.643G→A(p.R215Q;在两名患者中)和外显子8 c.1218G→A(p.A407T)。这些突变的合并频率在APL患者中为0.222,而在1100名受试者的多民族对照样本中为0.0018(P = 2.1×10 -7),在330名白人对照样本中为0.0045(P = 1.2×10 -5)。这些新的杂合突变是首次报道的LMNB2突变,也是首次在APL患者中报道,表明对重新注释的候选基因进行测序如何能够揭示新的疾病相关突变。