恶性肿瘤的基因治疗:挑战与希望并存

Masami Fujita
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摘要

恶性肿瘤基因治疗研究是一个快速发展的领域,在抗癌斗争中显示出巨大的潜力。虽然它有能力取得成功,但应对复杂多样的肿瘤生物学仍然是一个巨大的障碍。为解决这一问题,人们研究了多种方法,包括使用基于病毒载体的基因递送系统、纳米粒子和电穿孔等非病毒技术以及 CRISPR-Cas9 等基因组编辑技术。这些疗法试图改变肿瘤细胞的遗传物质,以阻碍其生长或引发细胞死亡。然而,要在临床环境中正常实施基因疗法,还必须克服大量障碍。这些因素包括确保精确靶向、尽量减少意外后果、优化治疗基因在肿瘤细胞中的转运以及控制对所用载体的免疫反应。为了克服这些问题,有必要建立跨学科联盟,并开展全面的临床前研究,以保证安全性和有效性。然而,由于分子生物学和基因编辑技术的巨大进步,基因疗法作为一种有效治疗恶性肿瘤的新方法,具有巨大的潜力。
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Gene Therapy of Malignant Tumor: A Challenge but a Promising Way
The study of gene therapy for malignant tumors is a rapidly developing area that shows great potential in the battle against cancer. Although it has the capacity for success, tackling the complexity and variety of tumor biology remains a substantial obstacle. Several approaches have been investigated to address this problem, including the use of viral vector-based gene delivery systems, non-viral techniques such as nanoparticles and electroporation, and genome editing technologies like CRISPR-Cas9. These treatments seek to alter the genetic material of tumor cells in order to either impede their growth or trigger cell death. Nevertheless, there exist substantial obstacles that must be surmounted prior to the regular implementation of gene therapy in clinical settings. These factors include assuring precise targeting, minimizing unintended consequences, optimizing the transport of therapeutic genes into tumor cells, and controlling immune responses to the vectors used. To overcome these problems, it will be necessary to establish interdisciplinary alliances and conduct thorough preclinical research to guarantee both safety and effectiveness. However, due to the tremendous progress in molecular biology and gene editing technology, gene therapy has significant potential as a new method for effectively treating malignant tumors.
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