Botschner J, Smith D W, Simell O, Scriver C R
Medical Research Council Genetics Group, McGill University-Montreal Children's Hospital, Quebec, Canada.
J Inherit Metab Dis. 1989;12(1):33-40. doi: 10.1007/BF01805528.
We measured L-ornithine oxidation in cultured skin fibroblasts from seven patients with hyperornithinaemia-hyperammonaemia-homocitrullinuria (HHH) syndrome (McKusick 23897), and compared it with oxidation by ornithine aminotransferase deficient gyrate atrophy (McKusick 25887) cells and lysinuric protein intolerance (McKusick 22270) cells in which there is an ornithine transport abnormality at the plasma membrane. Net uptake of ornithine is not abnormal in intact HHH cells. Ornithine oxidation was depressed in HHH and gyrate atrophy cells but not in lysinuric protein intolerance cells; the latter finding suggests there is no significant mitochondrial defect in lysinuric protein intolerance cells. Since HHH cells have intact ornithine aminotransferase, impaired oxidation is compatible with deficient penetration of ornithine into mitochondria in this disease. We could not demonstrate a gene dosage effect in oxidation values.
我们检测了7例高鸟氨酸血症-高氨血症-同型瓜氨酸尿症(HHH)综合征(麦库西克编号23897)患者培养的皮肤成纤维细胞中L-鸟氨酸的氧化情况,并将其与鸟氨酸转氨酶缺乏的回旋状萎缩症(麦库西克编号25887)细胞以及赖氨酸尿性蛋白不耐受症(麦库西克编号22270)细胞中的氧化情况进行了比较,后两种细胞在质膜存在鸟氨酸转运异常。完整的HHH细胞中鸟氨酸的净摄取并无异常。HHH细胞和回旋状萎缩症细胞中的鸟氨酸氧化受到抑制,但赖氨酸尿性蛋白不耐受症细胞中未受抑制;后一发现表明赖氨酸尿性蛋白不耐受症细胞中不存在明显的线粒体缺陷。由于HHH细胞具有完整的鸟氨酸转氨酶,氧化受损与该疾病中鸟氨酸进入线粒体的穿透不足相符。我们未能在氧化值中证明基因剂量效应。