Department of Biological Sciences and Bioengineering, Indian Institute of Technology, Kanpur, UP, India.
The Mehta Family Centre for Engineering in Medicine, Indian Institute of Technology, Kanpur, UP, India.
Cancer Gene Ther. 2022 May;29(5):402-417. doi: 10.1038/s41417-021-00331-7. Epub 2021 Apr 15.
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.
了解疾病的分子基础和设计合理设计的分子疗法一直是人类癌症治疗的圣杯。基于基因的疗法是实现可能治愈的重要途径。在过去三十年的集中研究中,为优化癌症基因治疗的潜力提供了重要线索。开发具有高潜力的基因疗法,以尽可能低的有效剂量杀死靶细胞,开发具有显著靶向癌症相关抗原能力的载体,应用辅助疗法靶向失调的 microRNA,并以疾病特异性的方式采用基因治疗和低剂量化疗相结合的混合策略将是至关重要的。本文概述了该领域的进展和挑战,重点介绍了用于基因转移的载体的生物学和范围、新确定的靶点及其在临床前和临床研究中的结果。