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[心血管分子遗传学与基因治疗。术语和技术介绍]

[Cardiovascular molecular genetics and gene therapy. Introduction to the terminology and technique].

作者信息

Eppenberger H M

机构信息

Institut für Zellbiologie der ETH Zürich.

出版信息

Schweiz Med Wochenschr. 1996 Oct 12;126(41):1716-20.

PMID:8966504
Abstract

The possible application of somatic gene therapy to the treatment of cardiovascular diseases is introduced; methods and delivery systems for transferring genes to the sites of application are discussed. Although cardiovascular diseases are mainly systemic disorders, critical lesions are frequently located at specific sites in the circulation and therefore accessible to the introduction of recombinant genes which may encode for proteins either inhibiting cell proliferation or promoting cell division. Cell-mediated gene transfer is compared to direct gene transfer in vivo; the advantages and disadvantages of the "ex vivo-in vitro-in vivo" and "direct in vivo" techniques are described. Several alternative gene transfer techniques are known. They include viral and retroviral vectors which have been made replication defective by genetic engineering, as well as non-viral systems such as DNA-liposome complexes, and physical methods such as the so-called biolistic technology originally developed to penetrate plant cell membranes. In summary, it is pointed out that even demonstrations of the few successful therapeutic applications to date do not conceal the fact that intensive experimental research using animal models is needed to prove the advantages and safety of the techniques used in gene therapy of the cardiovascular system.

摘要

介绍了体细胞基因治疗在心血管疾病治疗中的可能应用;讨论了将基因转移到应用部位的方法和递送系统。尽管心血管疾病主要是全身性疾病,但关键病变常常位于循环系统的特定部位,因此可通过导入重组基因来进行治疗,这些重组基因可编码抑制细胞增殖或促进细胞分裂的蛋白质。将细胞介导的基因转移与体内直接基因转移进行了比较;描述了“体外-体内-体外”和“直接体内”技术的优缺点。已知几种替代基因转移技术。它们包括通过基因工程使其复制缺陷的病毒和逆转录病毒载体,以及非病毒系统,如DNA-脂质体复合物,还有物理方法,如最初为穿透植物细胞膜而开发的所谓基因枪技术。总之,指出即使是迄今为止少数成功治疗应用的实例也掩盖不了这样一个事实,即需要利用动物模型进行深入的实验研究,以证明心血管系统基因治疗中所用技术的优势和安全性。

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