在X连锁肾上腺脑白质营养不良发病机制早期,ABCD4和BG1基因表达降低。
Decreased expression of ABCD4 and BG1 genes early in the pathogenesis of X-linked adrenoleukodystrophy.
作者信息
Asheuer Muriel, Bieche Ivan, Laurendeau Ingrid, Moser Ann, Hainque Bernard, Vidaud Michel, Aubourg Patrick
机构信息
Institut National de la Santé et de la Recherche Médicale U561, Hôpital Saint-Vincent de Paul, Paris, France.
出版信息
Hum Mol Genet. 2005 May 15;14(10):1293-303. doi: 10.1093/hmg/ddi140. Epub 2005 Mar 30.
Childhood cerebral adrenoleukodystrophy (CCER), adrenomyeloneuropathy (AMN) and AMN with cerebral demyelination (AMN-C) are the main phenotypic variants of X-linked adrenoleukodystrophy (ALD). It is caused by mutations in the ABCD1 gene encoding a half-size peroxisomal transporter that has to dimerize to become functional. The biochemical hallmark of ALD is the accumulation of very-long-chain fatty acids (VLCFA) in plasma and tissues. However, there is no correlation between the ALD phenotype and the ABCD1 gene mutations or the accumulation of VLCFA in plasma and fibroblast from ALD patients. The absence of genotype-phenotype correlation suggests the existence of modifier genes. To elucidate the mechanisms underlying the phenotypic variability of ALD, we studied the expression of ABCD1, three other peroxisomal transporter genes of the same family (ABCD2, ABCD3 and ABCD4) and two VLCFA synthetase genes (VLCS and BG1) involved in VLCFA metabolism, as well as the VLCFA concentrations in the normal white matter (WM) from ALD patients with CCER, AMN-C and AMN phenotypes. This study shows that: (1) ABCD1 gene mutations leading to truncated ALD protein are unlikely to cause variation in the ALD phenotype; (2) accumulation of saturated VLCFA in normal-appearing WM correlates with ALD phenotype and (3) expression of the ABCD4 and BG1, but not of the ABCD2, ABCD3 and VLCS genes, tends to be correlated with the severity of the disease, acting early in the pathogenesis of ALD.
儿童脑型肾上腺脑白质营养不良(CCER)、肾上腺脊髓神经病(AMN)以及伴有脑脱髓鞘的AMN(AMN-C)是X连锁肾上腺脑白质营养不良(ALD)的主要表型变异。它由编码一种半尺寸过氧化物酶体转运蛋白的ABCD1基因突变引起,该转运蛋白必须二聚化才能发挥功能。ALD的生化特征是血浆和组织中极长链脂肪酸(VLCFA)的积累。然而,ALD表型与ABCD1基因突变或ALD患者血浆和成纤维细胞中VLCFA的积累之间没有相关性。基因型-表型相关性的缺失表明存在修饰基因。为了阐明ALD表型变异的潜在机制,我们研究了ABCD1、同一家族的其他三个过氧化物酶体转运蛋白基因(ABCD2、ABCD3和ABCD4)以及参与VLCFA代谢的两个VLCFA合成酶基因(VLCS和BG1)的表达,以及患有CCER、AMN-C和AMN表型的ALD患者正常白质(WM)中的VLCFA浓度。这项研究表明:(1)导致截短的ALD蛋白的ABCD1基因突变不太可能引起ALD表型的变异;(2)外观正常的WM中饱和VLCFA的积累与ALD表型相关,并且(3)ABCD4和BG1基因的表达,而不是ABCD2、ABCD3和VLCS基因的表达,往往与疾病严重程度相关,在ALD发病机制中起早期作用。