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首页> 外文期刊>Current opinion in lipidology >Recent advances in liver-directed gene therapy: implications for the treatment of dyslipidemia.
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Recent advances in liver-directed gene therapy: implications for the treatment of dyslipidemia.

机译：肝定向基因治疗的最新进展：对血脂异常的治疗意义。

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摘要

Somatic gene therapy for the treatment of dyslipidemia is an area of active investigation. A substantial body of data indicates that the transfer of various lipid-lowering genes to the liver is an effective method of restoring normal plasma lipids in animal models of dyslipidemia. Most studies have used adenoviral vectors because of their excellent gene-transfer efficiency. However, the first and second-generation adenoviral vectors used in these experiments are highly toxic and are associated with substantial morbidity and mortality. This article reviews current data on the properties of two novel vectors, the adeno-associated virus and the helper-dependent adenovirus that is devoid of all protein-encoding genes. Each type of vector has its advantages and drawbacks. They appear to be the most promising vectors to date for liver-directed gene transfer in the treatment of dyslipidemia.
机译：体细胞基因疗法治疗血脂异常是积极研究的领域。大量数据表明，各种血脂降低基因向肝脏的转移是在血脂异常动物模型中恢复正常血浆脂质的有效方法。大多数研究都使用腺病毒载体，因为它们具有出色的基因转移效率。然而，在这些实验中使用的第一代和第二代腺病毒载体是剧毒的，并与大量发病率和死亡率有关。本文回顾了目前关于两种新型载体的特性的数据，这两种新型载体是腺相关病毒和缺乏所有蛋白质编码基因的辅助依赖型腺病毒。每种类型的载体都有其优点和缺点。它们似乎是迄今为止用于血脂异常的肝定向基因转移的最有前景的载体。

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