目前，采用标准治疗化疗、放疗和/或免疫疗法治疗实体肿瘤通常受到严重的不良毒副作用的限制，导致治疗指数较窄。癌症基因治疗代表着一种有针对性的方法，原则上可以显著减少正常组织中的不良副作用，同时显著抑制肿瘤的生长和进展。为了达到有效的效果，这种策略需要对肿瘤发展和进化的分子生物学有清晰的理解，并且需要开发可以作为靶向癌细胞载体的生物向量。组学技术的出现和优化，允许在定义的复杂生物样品中对基因（基因组学）、mRNA（转录组学）、蛋白质（蛋白质组学）、代谢物（代谢组学）、表观基因组学（表观遗传学、表观转录组学和表观蛋白质组学）及其相互作用进行集体和空间的识别，为确定癌症范式相关的关键靶点提供了一条路线。将这些策略与控制靶基因表达的已确定的遗传元素相结合，揭示了为癌症开发有指导性的基因治疗提供重要机会。本综述的目的是概述开发基因启动子导向的靶向表达关键基因的当前状态和潜在限制，并强调它们在癌症基因治疗中的潜在应用。版权所有 © 2023。由Elsevier Inc.发表。

Current treatment of solid tumors with standard of care chemotherapies, radiation therapy and/or immunotherapies are often limited by severe adverse toxic effects, resulting in a narrow therapeutic index. Cancer gene therapy represents a targeted approach that in principle could significantly reduce undesirable side effects in normal tissues while significantly inhibiting tumor growth and progression. To be effective, this strategy requires a clear understanding of the molecular biology of cancer development and evolution and developing biological vectors that can serve as vehicles to target cancer cells. The advent and fine tuning of omics technologies that permit the collective and spatial recognition of genes (genomics), mRNAs (transcriptomics), proteins (proteomics), metabolites (metabolomics), epiomics (epigenomics, epitranscriptomics, and epiproteomics), and their interactomics in defined complex biological samples provide a roadmap for identifying crucial targets of relevance to the cancer paradigm. Combining these strategies with identified genetic elements that control target gene expression uncovers significant opportunities for developing guided gene-based therapeutics for cancer. The purpose of this review is to overview the current state and potential limitations in developing gene promoter-directed targeted expression of key genes and highlights their potential applications in cancer gene therapy.Copyright © 2023. Published by Elsevier Inc.