Max-Delbrück-Center for Molecular Medicine, Berlin, Germany.
Experimental and Clinical Research Center, Charité - Universitätsmedizin Berlin, Berlin, Germany.
Methods Mol Biol. 2022;2521:1-21. doi: 10.1007/978-1-0716-2441-8_1.
Gene therapy has started in the late 1980s as novel, clinically applicable therapeutic option. It revolutionized the treatment of genetic diseases with the initial intent to repair or replace defective genes. Gene therapy has been adapted for treatment of malignant diseases to improve the outcome of cancer patients. In fact, cancer gene therapy has rapidly gained great interest and evolved into a research field with highest proportion of research activities in gene therapy. In this context, cancer gene therapy has long entered translation into clinical trials and therefore more than two-thirds of all gene therapy trials worldwide are aiming at the treatment of cancer disease using different therapeutic strategies. During the decades in cancer gene therapy, tremendous knowledge has accumulated. This led to significant improvements in vector design, transgene repertoire, more targeted interventions, use of novel gene therapeutic technologies such as CRISPR/Cas, sleeping beauty vectors, and development of effective cancer immunogene therapies. In this chapter, a brief overview of current key developments in cancer gene therapy is provided to gain insights into the recent directions in research as well as in clinical application of cancer gene therapy.
基因治疗始于 20 世纪 80 年代后期,作为一种新颖的、临床适用的治疗选择。它最初旨在修复或替换有缺陷的基因,从而彻底改变了遗传疾病的治疗方法。基因治疗已被用于治疗恶性疾病,以改善癌症患者的预后。事实上,癌症基因治疗已经迅速引起了极大的关注,并发展成为基因治疗中研究活动比例最高的研究领域。在这种情况下,癌症基因治疗早已进入临床试验阶段,因此,全球超过三分之二的基因治疗试验旨在使用不同的治疗策略治疗癌症疾病。在癌症基因治疗的几十年中,已经积累了大量的知识。这导致载体设计、转基因库、更靶向的干预措施、新型基因治疗技术(如 CRISPR/Cas、睡美人载体)的使用以及有效的癌症免疫基因治疗的发展方面都取得了显著的进展。在本章中,简要概述了癌症基因治疗的当前关键进展,以深入了解癌症基因治疗的研究方向和临床应用。