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癌症基因治疗:临床试验

Cancer gene-therapy: clinical trials.

作者信息

Palmer Daniel H, Young Lawrence S, Mautner Vivien

机构信息

CR UK Institute for Cancer Studies, University of Birmingham B15 2TT, UK.

出版信息

Trends Biotechnol. 2006 Feb;24(2):76-82. doi: 10.1016/j.tibtech.2005.12.002. Epub 2005 Dec 27.

Abstract

The objective of gene therapy for the treatment of cancer is to kill tumour cells but preserve normal tissue; therefore, the ideal gene therapy agent would be targeted for specific transduction of tumour cells and have specificity in its cytotoxic action. A variety of strategies to achieve these aims have demonstrated promising results in the laboratory, including enzyme-pro-drug activating systems, correction of genetic mutations contributing to the malignant phenotype and stimulation of a T-cell-mediated anti-tumour immune response. The key to the success of all these strategies is an effective vector that can direct appropriate expression of the therapeutic gene. Viruses have many properties that can be adapted to achieve this therapeutic endpoint; furthermore, they can be engineered to replicate selectively in cancer cells and lyse them. The challenge now is to translate these features into effective therapies that can supplement or supplant existing treatments.

摘要

癌症基因治疗的目标是杀死肿瘤细胞同时保留正常组织;因此,理想的基因治疗药物应靶向肿瘤细胞进行特异性转导,并在细胞毒性作用方面具有特异性。为实现这些目标的各种策略已在实验室中显示出有希望的结果,包括酶-前药激活系统、纠正导致恶性表型的基因突变以及刺激T细胞介导的抗肿瘤免疫反应。所有这些策略成功的关键是一种能够指导治疗基因适当表达的有效载体。病毒具有许多可被改造以实现这一治疗终点的特性;此外,它们可以被设计成在癌细胞中选择性复制并裂解癌细胞。现在的挑战是将这些特性转化为能够补充或替代现有治疗方法的有效疗法。

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