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辅助性肝移植:选择性代谢紊乱中的一种基因治疗形式。

Auxiliary liver transplantation: a form of gene therapy in selective metabolic disorders.

作者信息

Shanmugam Naresh P, Perumalla Rajasekhar, Gopinath Rg, Olithselvan A, Varghese Joy, Kapoor Dharmesh, Rela Mohmed

机构信息

Institute of Liver Disease and Transplantation, Global Hospitals and Health City, Chennai, Tamil Nadu, India.

出版信息

J Clin Exp Hepatol. 2011 Sep;1(2):118-20. doi: 10.1016/S0973-6883(11)60132-1. Epub 2011 Nov 9.

Abstract

Auxiliary liver transplantation is an accepted form of therapy in acute liver failure and in certain metabolic disorders. We report India's first successful auxiliary liver transplantation for Crigler-Najjar syndrome type 1, showing that it is technically feasible and safe procedure. It is utmost important to select appropriate cases for auxiliary transplant for successful long-term outcome. The surgeon should also have an understanding of the portal flow dynamics, as steal phenomenon can occur, depriving blood blow to either graft or native liver. Though successful in animal models, gene therapy is still in experimental stage in humans and pace of progress has been disappointing. Auxiliary liver transplantation retains the native liver for future gene therapy. These children are young and are likely to have a long life expectancy, and withdrawal of immunosuppression would be a huge advantage.

摘要

辅助性肝移植是治疗急性肝衰竭和某些代谢紊乱的一种公认疗法。我们报告了印度首例成功的针对1型克里格勒-纳贾尔综合征的辅助性肝移植,表明这是一种技术上可行且安全的手术。为获得成功的长期疗效,选择合适的辅助性移植病例至关重要。外科医生还应了解门静脉血流动力学,因为可能会出现盗血现象,导致移植肝或原生肝供血不足。尽管基因疗法在动物模型中取得了成功,但在人类中仍处于实验阶段,进展速度令人失望。辅助性肝移植保留了原生肝以备未来进行基因治疗。这些儿童年龄尚小,预期寿命较长,停用免疫抑制将是一个巨大优势。

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