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肝豆状核变性治疗的研究近况

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摘要 肝豆状核变性(WD)是一种遗传性铜代谢障碍性疾病,表现为铜异常沉积而致多脏器损伤。WD致病基因主要在肝脏表达。提供患者正常的肝细胞(或组织)可以纠正其遗传缺陷,改善铜代谢。已知多种药物对WD患者的治疗有效,但存在长期用药的不良反应和影响患者生活质量的问题。肝(干)细胞治疗WD目前研究较多,有可能成为最有前途的WD治疗方法。
作者 阴瑞红 林森
出处 《世界临床药物》 CAS 2006年第12期735-738,共4页 World Clinical Drug
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参考文献18

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