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肺部疾病的基因治疗。

Gene therapy for lung disease.

作者信息

Ennist D L

机构信息

Respiratory Gene Therapy Program, Genetic Therapy Inc., a Novartis Company, 938 Clopper Road, Gaithersburg, MD 20878, USA.

出版信息

Trends Pharmacol Sci. 1999 Jun;20(6):260-6. doi: 10.1016/s0165-6147(99)01350-4.

Abstract

Gene therapy is a new field of medical research that has great potential to influence the course of treatment of human disease. The lung has been a particularly attractive target organ for gene therapy due to its accessibility and the identification of genetic deficits for a number of lung diseases. Several clinical trials have shown evidence of low levels of gene transfer and expression, but without any benefit to the patients involved. Thus, current studies are focusing on further research and technological improvements to the vectors. Gene therapy is now beginning to benefit from a shift in emphasis from clinical trials to the development of better tools and procedures to deliver gene therapy to the bedside.

摘要

基因治疗是医学研究的一个新领域,极有可能影响人类疾病的治疗进程。由于肺部易于触及,且已确定了多种肺部疾病的基因缺陷,因此肺部一直是基因治疗特别有吸引力的靶器官。多项临床试验显示了基因转移和表达水平较低的证据,但对相关患者并无任何益处。因此,目前的研究集中在对载体的进一步研究和技术改进上。基因治疗现在开始受益于重点的转变,即从临床试验转向开发更好的工具和程序,以便将基因治疗应用于临床。

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