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线粒体脂肪酸β-氧化遗传性酶缺乏小鼠中的协同杂合性

Synergistic heterozygosity in mice with inherited enzyme deficiencies of mitochondrial fatty acid beta-oxidation.

作者信息

Schuler A Michele, Gower Barbara A, Matern Dietrich, Rinaldo Piero, Vockley Jerry, Wood Philip A

机构信息

Department of Genetics, University of Alabama at Birmingham, Birmingham, AL, USA.

出版信息

Mol Genet Metab. 2005 May;85(1):7-11. doi: 10.1016/j.ymgme.2004.09.006. Epub 2005 Feb 16.

Abstract

We have used mice with inborn errors of mitochondrial fatty acid beta-oxidation to test the concept of synergistic heterozygosity. We postulated that clinical disease can result from heterozygous mutations in more than one gene in single or related metabolic pathways. Mice with combinations of mutations in mitochondrial fatty acid beta-oxidation genes were cold challenged to test their ability to maintain normal body temperature, a sensitive indicator of overall beta-oxidation function. This included mice of the following genotypes: triple heterozygosity for mutations in very-long-chain acyl CoA dehydrogenase, long-chain acyl CoA dehydrogenase, and short-chain acyl CoA dehydrogenase genes (VLCAD+/-//LCAD+/-//SCAD+/-); double heterozygosity for mutations in VLCAD and LCAD genes (VLCAD+/-//LCAD+/-); double heterozygosity for mutations in LCAD and SCAD genes (LCAD+/-//SCAD+/-); single heterozygous mice (VLCAD+/-, LCAD+/-, SCAD+/-) and wild-type. We found that approximately 33% of mice with any of the combined mutant genotypes tested became hypothermic during a cold challenge. All wild-type and single heterozygous mice maintained normal body temperature throughout a cold challenge. Despite development of hypothermia in some double heterozygous mice, blood glucose concentrations remained normal. Biochemical screening by acylcarnitine and fatty acid analyses demonstrated results that varied by genotype. Thus, physiologic reduction of the beta-oxidation pathway, characterized as cold intolerance, occurred in mice with double or triple heterozygosity; however, the derangement was milder than in mice homozygous for any of these mutations. These results substantiate the concept of synergistic heterozygosity and illustrate the potential complexity involved in diagnosis and characterization of inborn errors of fatty acid metabolism in humans.

摘要

我们利用患有线粒体脂肪酸β氧化先天性缺陷的小鼠来测试协同杂合性的概念。我们推测,临床疾病可能由单个或相关代谢途径中多个基因的杂合突变引起。对线粒体脂肪酸β氧化基因突变组合的小鼠进行冷刺激,以测试它们维持正常体温的能力,这是整体β氧化功能的一个敏感指标。这包括以下基因型的小鼠:极长链酰基辅酶A脱氢酶、长链酰基辅酶A脱氢酶和短链酰基辅酶A脱氢酶基因突变的三重杂合子(VLCAD+/-//LCAD+/-//SCAD+/-);VLCAD和LCAD基因突变的双重杂合子(VLCAD+/-//LCAD+/-);LCAD和SCAD基因突变的双重杂合子(LCAD+/-//SCAD+/-);单杂合小鼠(VLCAD+/ -、LCAD+/ -、SCAD+/-)和野生型。我们发现,在接受测试的任何组合突变基因型的小鼠中,约33%在冷刺激期间出现体温过低。所有野生型和单杂合小鼠在整个冷刺激过程中都保持正常体温。尽管一些双重杂合小鼠出现了体温过低,但血糖浓度仍保持正常。通过酰基肉碱和脂肪酸分析进行的生化筛查显示,结果因基因型而异。因此,在双重或三重杂合的小鼠中出现了以不耐寒为特征的β氧化途径的生理性降低;然而,这种紊乱比这些突变的纯合小鼠要轻。这些结果证实了协同杂合性的概念,并说明了人类脂肪酸代谢先天性缺陷的诊断和特征描述中潜在的复杂性。

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