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X-linked adrenoleukodystrophy gene and corresponding protein
X-linked adrenoleukodystrophy gene and corresponding protein
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机译:X连锁肾上腺白质营养不良基因和相应的蛋白质
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摘要
A method for the treatment of adrenoleukodystrophy or adrenomyeloneuropathy, in which there is administered to a human patient in need of the same an effective amount of hematopoietic cells modified ex vivo by infection with a retroviral vector containing a nucleic acid fragment comprising a sequence as represented on FIG. 2 (SEQ. ID No: 1) or FIG. 6 (SEQ. ID. No: 4-17) or FIG. 7 (SEQ. ID No: 18-23). Also, human hematopoietic cells can be treated ex vivo to correct the content of very long chain fatty acids in them, by providing human hematopoietic cells of a patient affected by adrenoleukodystrophy or adrenomyeloneuropathy, and infecting them ex vivo with a retroviral vector containing, operably linked to a promoter, a nucleic acid fragment as above. The expression of the nucleic acid in the hematopoietic cells corrects the content of very long chain fatty acids in the hematopoietic cells.
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机译:一种治疗肾上腺皮质营养不良或肾上腺皮质神经病的方法,其中向需要同样剂量的人有效剂量的人用经逆转录病毒载体感染的活体外造血细胞进行体外修饰,所述逆转录病毒载体含有含有如上代表的序列的核酸片段。图。图2(SEQ ID NO:1)或图2。图6(SEQ.ID.No.:4-17)或图6。 7(SEQ。ID No:18-23)。而且,可以通过提供受肾上腺白细胞营养不良或肾上腺髓质神经病影响的患者的人类造血细胞,并用含有可操作地连接的逆转录病毒载体离体感染来对造血细胞进行离体治疗,以纠正其中的长链脂肪酸含量。对于启动子,为上述核酸片段。核酸在造血细胞中的表达纠正了造血细胞中非常长链脂肪酸的含量。
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