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遗传性代谢疾病的器官移植

Organ transplantation for inherited metabolic disease.

作者信息

Kelly D A

机构信息

Liver Unit, Children's Hospital, Ladywood Middleway, Birmingham.

出版信息

Arch Dis Child. 1994 Sep;71(3):181-3. doi: 10.1136/adc.71.3.181.

DOI:10.1136/adc.71.3.181
PMID:7979487
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC1029967/
Abstract

Liver transplantation for inherited metabolic disease is a therapeutic reality in the 1990s. Careful patient selection and timing and choice of operative procedure should greatly improve the quality of life and long term survival for those children with life threatening metabolic disease. The rapid expansion of molecular genetics and the development of effective gene therapy may well displace liver transplantation as appropriate treatment of these disorders in the future.

摘要

肝脏移植用于治疗遗传性代谢疾病在20世纪90年代已成为现实。仔细挑选患者、把握手术时机以及选择手术方式,对于那些患有危及生命的代谢疾病的儿童而言,应能极大地改善其生活质量并延长长期生存率。分子遗传学的迅速发展以及有效基因疗法的开发,很可能在未来取代肝脏移植成为这些疾病的合适治疗手段。

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Organ transplantation for inherited metabolic disease.遗传性代谢疾病的器官移植
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本文引用的文献

1
Liver transplantation in newborn liver failure: treatment for neonatal hemochromatosis.新生儿肝衰竭的肝移植:新生儿血色病的治疗
Transplant Proc. 1993 Feb;25(1 Pt 2):1068-71.
2
Orthotopic liver transplantation in two adults with Niemann-Pick and Gaucher's diseases: implications for the treatment of inherited metabolic disease.
Hepatology. 1993 Jan;17(1):42-9.
3
Successful liver transplantation in babies under 1 year.1岁以下婴儿的成功肝脏移植
BMJ. 1993 Oct 2;307(6908):825-8. doi: 10.1136/bmj.307.6908.825.
4
Orthotopic liver transplantation for hepatic complications of Wilson's disease.用于威尔逊病肝脏并发症的原位肝移植
Br J Surg. 1993 Jul;80(7):909-11. doi: 10.1002/bjs.1800800738.
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Intractable neurological Wilson's disease treated with orthotopic liver transplantation.原位肝移植治疗难治性神经型威尔逊病。
Dig Dis Sci. 1993 Sep;38(9):1746-50. doi: 10.1007/BF01303186.
6
Auxiliary liver grafting: a new concept in liver transplantation.辅助性肝移植:肝移植中的一个新概念。
Lancet. 1993 Sep 25;342(8874):758. doi: 10.1016/0140-6736(93)91537-v.
7
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Lancet. 1993 Sep 25;342(8874):779-80. doi: 10.1016/0140-6736(93)91543-u.
8
Liver transplantation for advanced liver disease with alpha-1-antitrypsin deficiency.针对患有α-1抗胰蛋白酶缺乏症的晚期肝病进行肝移植。
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9
Orthotopic liver transplantation for type I Crigler-Najjar syndrome.原位肝移植治疗I型克里格勒-纳贾尔综合征
Hepatology. 1986 Nov-Dec;6(6):1259-62. doi: 10.1002/hep.1840060606.
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Liver transplantation in cystic fibrosis.
Lancet. 1989 May 13;1(8646):1073. doi: 10.1016/s0140-6736(89)92464-1.