了解肝细胞癌的基因参与:对基因疗法和个性化医疗的启示

Mahmoud A Younis, Hideyoshi Harashima
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摘要

摘要:肝细胞癌(HCC)是肝癌的主要类型,也是全球最致命的健康威胁之一。传统的 HCC 治疗方案因效率低和难以忍受的副作用而受到阻碍。然而,基因疗法现在为治疗许多以前被认为是不治之症的疾病带来了希望,基因疗法正开始解决传统疗法的许多不足之处。在此,我们总结了基因在 HCC 发病机制和预后中的参与情况,尤其关注信号通路失调、参与免疫逃避的基因以及作为新型双刃剑的非编码 RNA,它们共同为 HCC 的基因治疗提供了潜在靶点。本文讨论了 HCC 基因治疗的机遇与挑战。其中包括基因组编辑和细胞疗法等创新疗法。此外,还重点介绍了将纳米药物用于 HCC 基因治疗的先进基因递送技术。关键词:肝细胞癌;基因治疗;个性化医疗;纳米药物;临床转化
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Understanding Gene Involvement in Hepatocellular Carcinoma: Implications for Gene Therapy and Personalized Medicine
Abstract: Hepatocellular carcinoma (HCC) is the dominant type of liver cancers and is one of the deadliest health threats globally. The conventional therapeutic options for HCC are hampered by low efficiency and intolerable side effects. Gene therapy, however, now offers hope for the treatment of many disorders previously considered incurable, and gene therapy is beginning to address many of the shortcomings of conventional therapies. Herein, we summarize the involvement of genes in the pathogenesis and prognosis of HCC, with a special focus on dysregulated signaling pathways, genes involved in immune evasion, and non-coding RNAs as novel two-edged players, which collectively offer potential targets for the gene therapy of HCC. Herein, the opportunities and challenges of HCC gene therapy are discussed. These include innovative therapies such as genome editing and cell therapies. Moreover, advanced gene delivery technologies that recruit nanomedicines for use in gene therapy for HCC are highlighted. Finally, suggestions are offered for improved clinical translation and future directions in this area of endeavor.

Keywords: hepatocellular carcinoma, gene therapy, personalized medicine, nanomedicines, clinical translation
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