基因治疗的发展现状、问题和展望

摘　要：基因治疗是一种新的治疗手段，可以治疗多种疾病，包括癌症、遗传性疾病、感染性疾病、心血管疾病和自身免疫性疾病。癌症基因治疗是基因治疗的主要应用领域。过去几年里，全球基因治疗临床试验取得了很大的进步。实际上，基因治疗也遇到了很多困难。未来，基因治疗的主要目标是发展安全和高效的基因导入系统，它们能将外源遗传物质靶向性地导入到特异的细胞。本文主要综述基因治疗所取得的突出进展、所遇到的困难和发展前景。

关键词：基因治疗；导入系统；安全；高效

Current status, problems and prospects in gene therapy

Abstract: Gene therapy is a new therapeutic tool for the treatment of diverse types of diseases, including cancer，congenital genetic, infectious, cardiovascular and autoimmune diseases. Cancer gene therapy is the most studied application of gene therapy. In the last few years, significant advances have been made in the clinical trials worldwide. In practice, considerable obstacles have emerged. In the future, the main objective in gene therapy is the development of safe and high efficient gene delivery systems that can deliver foreign genetic materials into specific cell types. This article reviews the most recent studies highlighting the advances, the problems and future prospects of gene therapy.

Key words: gene therapy; delivery systems; safe; high efficient