肝脏代谢先天性缺陷的基因治疗：迈向临床应用的进展。
Gene therapy for inborn errors of liver metabolism: progress towards clinical applications.
机构信息
Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.
出版信息
Ital J Pediatr. 2008 Nov 18;34(1):2. doi: 10.1186/1824-7288-34-2.
Abstract
The treatment for inborn errors of liver metabolism is based on dietary, drug, and cell therapies (orthotopic liver transplantation). However, significant morbidity and mortality still remain, and alternative strategies are needed. Gene replacement therapy has the potential of providing a definitive cure for patients with these diseases. Significant progress has been made in the pre-clinical arena and achievement of efficacy in different animal models has been reported using multiple gene transfer technologies. This article summarizes the gene transfer strategies being investigated, the pre-clinical data, and the available early clinical results for inborn errors of liver metabolism.
摘要
肝脏代谢先天性缺陷的治疗方法基于饮食、药物和细胞疗法（原位肝移植）。然而，仍然存在显著的发病率和死亡率，因此需要替代策略。基因替代疗法有可能为这些疾病的患者提供明确的治愈方法。在临床前领域已经取得了重大进展，并且已经报道了使用多种基因转移技术在不同动物模型中实现疗效的情况。本文总结了正在研究的基因转移策略、临床前数据以及肝脏代谢先天性缺陷的现有早期临床结果。
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