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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.

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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