Nucleic acid-based therapeutics like synthetic small interfering RNAs have been exploited to modulate gene function, taking advantage of RNA interference (RNAi), an evolutionally conserved biological process. Recently, the world's first RNAi drug was approved for a rare genetic disorder in the liver. However, there are significant challenges that need to be resolved before RNAi can be translated in other genetic diseases like cancer. Current drug delivery platforms for therapeutic silencing RNAs are tailored to hepatic targets. RNAi therapies for nonhepatic conditions are still at early clinical phases. In this study, we discuss the critical design considerations in anticancer RNAi drug development, insights gained from initial clinical trials, and new strategies that are entering clinical development, shaping the future of RNAi in cancer.

中文翻译：

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基于 RNA 干扰的癌症药物：障碍和承诺的“交付”。

基于核酸的疗法，如合成的小干扰 RNA，已被用来调节基因功能，利用 RNA 干扰 (RNAi)，一种进化上保守的生物过程。最近，世界上第一种 RNAi 药物被批准用于治疗一种罕见的肝脏遗传疾病。然而，在将 RNAi 转化为癌症等其他遗传疾病之前，需要解决一些重大挑战。当前用于治疗性沉默 RNA 的药物递送平台是针对肝脏靶点量身定制的。针对非肝脏疾病的 RNAi 疗法仍处于早期临床阶段。在这项研究中，我们讨论了抗癌 RNAi 药物开发中的关键设计考虑因素、从初始临床试验中获得的见解，以及正在进入临床开发、塑造癌症 RNAi 未来的新策略。