Adrenoleukodystrophy: the restoration of peroxisomal beta-oxidation by transfection of normal cDNA.
Adrenoleukodystrophy: the restoration of peroxisomal beta-oxidation by transfection of normal cDNA.
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肾上腺脑白质营养不良：通过转染正常 cDNA 恢复过氧化物酶体 β-氧化。
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发表时间：
1995
期刊：
影响因子：
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通讯作者：
Takashi Kobayashi
中科院分区：
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作者：
N. Shinnoh;Takeshi Yamada;T. Yoshimura;Hirokazu Furuya;Y. Yoshida;Yutaka Suzuki;N. Shimozawa;T. Orii;Takashi Kobayashi
为了阐明ALDP[一种由肾上腺脑白质营养不良（ALD）基因编码的蛋白质]的功能，将正常ALDP cDNA插入由鸡β -肌动蛋白启动子驱动的表达载体中，转染到ALD成纤维细胞中。在瞬时表达系统中，即使根据western blot分析结果新合成了ALDP，脂肪酸组成也没有改变。在稳定表达系统中，3株细胞株ALDP呈强阳性。在这些细胞中，超长链脂肪酸（C26:0）的水平与对照组一样低，而通过两种不同的方法检测，C24 β -氧化活性恢复正常。综上所述，ALDP对过氧化物酶体β -氧化的功能是必不可少的，因此ALDP的治疗可能是通过补充ALDP。
In order to elucidate the function of ALDP [a protein encoded by the gene responsible for adrenoleukodystrophy (ALD)], normal ALDP cDNA, inserted in an expression vector driven by chicken beta-actin promotor, was transfected into ALD fibroblasts. In a transient expression system, the fatty acid composition did not change even though the ALDP was newly synthesized based on the findings of a western blot analysis. In a stable expression system, 3 cell lines were strongly positive for ALDP. In these cells the level of very long chain fatty acid (C26:0) turned out to be as low as those of the control, while the activities of C24 beta-oxidation, as checked by two different methods, became normal. From these results, it is concluded that ALDP is indispensable for the function of peroxisomal beta-oxidation, and thus the treatment of ALD may be possible by the supplementation of ALDP.