文章：

载体工程，癌症基因治疗的策略和靶点

Vector engineering, strategies and targets in cancer gene therapy 

原文发布日期：2021-04-15 

英文摘要：

Understanding the molecular basis of disease and the design of rationally designed molecular therapies has been the holy grail in the management of human cancers. Gene-based therapies are an important avenue for achieving a possible cure. Focused research in the last three decades has provided significant clues to optimize the potential of cancer gene therapy. The development of gene therapies with a high potential to kill the target cells at the lowest effective dose possible, the development of vectors with significant ability to target cancer-associated antigen, the application of adjunct therapies to target dysregulated microRNA, and embracing a hybrid strategy with a combination of gene therapy and low-dose chemotherapy in a disease-specific manner will be pivotal. This article outlines the advances and challenges in the field with emphasis on the biology and scope of vectors used for gene transfer, newer targets identified, and their outcome in preclinical and clinical studies. 

摘要翻译： 

理解疾病的分子基础并设计合理靶向的分子疗法，一直是人类癌症治疗领域的终极目标。基因治疗为实现潜在治愈提供了重要途径。过去三十年的集中研究为优化癌症基因治疗潜力提供了重要线索：开发能够在最低有效剂量下高效杀灭靶细胞的基因治疗策略，构建具有显著癌症抗原靶向能力的载体系统，应用靶向调控异常microRNA的辅助疗法，以及采用基因治疗与低剂量化疗相结合的疾病特异性混合策略，都将成为关键突破点。本文综述了该领域的最新进展与挑战，重点探讨基因载体的生物学特性与应用前景、新发现的治疗靶点及其在临床前研究与临床试验中的成果。

原文链接：

Vector engineering, strategies and targets in cancer gene therapy