Abstract：
BACKGROUND: Small interference RNA (siRNA) has emerged as the most desired method for researchers and clinicians who wish to silence a specific gene of interest and has been extensively developed as a therapeutic agent. This review points to collecting all clinical trials on siRNA and understanding its benefits, pharmacokinetics and safety by reading articles published in the last 5 years.
METHODS: Searching in the PubMed database using \'siRNA\' and \'in vivo\' with limits to articles published in the previous 5 years, article type \'clinical trials\' and language \'English\' to acquire papers on in vivo studies on siRNA approaches. Features of siRNA clinical trials registered at https://clinicaltrials.gov/ were analysed.
RESULTS: So far, 55 clinical studies have been published on siRNA. Many published clinical trials on siRNA showed tolerability, safety and effectiveness in treating cancers like breast, lung, colon, and other organs and other diseases like viral infections and hereditary diseases. Many different routes of administration can silence many genes at the same time. Limitations and uncertainties associated with siRNA treatment include the effectiveness of cellular uptake, precise targeting of the intended tissue or cell and prompt elimination from the body.
CONCLUSIONS: The siRNA or RNAi method will be one of the most critical and influential techniques to fight against many different diseases. Although the RNAi approach has certain advantages, it also has limitations concerning clinical applications. Overcoming these limitations remains a daunting challenge.
METHODS: Searching in the PubMed database using \'siRNA\' and \'in vivo\' with limits to articles published in the previous 5 years, article type \'clinical trials\' and language \'English\' to acquire papers on in vivo studies on siRNA approaches. Features of siRNA clinical trials registered at https://clinicaltrials.gov/ were analysed.
RESULTS: So far, 55 clinical studies have been published on siRNA. Many published clinical trials on siRNA showed tolerability, safety and effectiveness in treating cancers like breast, lung, colon, and other organs and other diseases like viral infections and hereditary diseases. Many different routes of administration can silence many genes at the same time. Limitations and uncertainties associated with siRNA treatment include the effectiveness of cellular uptake, precise targeting of the intended tissue or cell and prompt elimination from the body.
CONCLUSIONS: The siRNA or RNAi method will be one of the most critical and influential techniques to fight against many different diseases. Although the RNAi approach has certain advantages, it also has limitations concerning clinical applications. Overcoming these limitations remains a daunting challenge.
摘要：
背景：小干扰RNA（siRNA）已成为希望沉默特定目的基因的研究人员和临床医生最理想的方法，并已被广泛开发为治疗剂。这篇综述指出收集所有关于siRNA的临床试验并了解其益处，通过阅读过去5年发表的文章，获得药代动力学和安全性。
方法：在PubMed数据库中使用\'siRNA\'和\'体内\'进行搜索，限制前5年发表的文章，文章类型\“临床试验\”和语言\“英语\”获得关于siRNA方法体内研究的论文。分析了在https://clinicaltrials.gov/注册的siRNA临床试验的特征。
结果：到目前为止，已经发表了55项关于siRNA的临床研究。许多已发表的关于siRNA的临床试验显示出耐受性，治疗乳腺癌等癌症的安全性和有效性，肺，结肠,以及其他器官和其他疾病，如病毒感染和遗传性疾病。许多不同的给药途径可以同时沉默许多基因。与siRNA治疗相关的局限性和不确定性包括细胞摄取的有效性，精确瞄准预期的组织或细胞，并迅速从体内消除。
结论：siRNA或RNAi方法将是对抗许多不同疾病的最关键和最有影响力的技术之一。尽管RNAi方法具有一定的优势,它也有关于临床应用的局限性。克服这些限制仍然是一项艰巨的挑战。
方法：在PubMed数据库中使用\'siRNA\'和\'体内\'进行搜索，限制前5年发表的文章，文章类型\“临床试验\”和语言\“英语\”获得关于siRNA方法体内研究的论文。分析了在https://clinicaltrials.gov/注册的siRNA临床试验的特征。
结果：到目前为止，已经发表了55项关于siRNA的临床研究。许多已发表的关于siRNA的临床试验显示出耐受性，治疗乳腺癌等癌症的安全性和有效性，肺，结肠,以及其他器官和其他疾病，如病毒感染和遗传性疾病。许多不同的给药途径可以同时沉默许多基因。与siRNA治疗相关的局限性和不确定性包括细胞摄取的有效性，精确瞄准预期的组织或细胞，并迅速从体内消除。
结论：siRNA或RNAi方法将是对抗许多不同疾病的最关键和最有影响力的技术之一。尽管RNAi方法具有一定的优势,它也有关于临床应用的局限性。克服这些限制仍然是一项艰巨的挑战。
