化学修饰的小干扰RNA的治疗潜力：近期趋势
Therapeutic potential of chemically modified siRNA: Recent trends.
作者信息
Selvam Chelliah, Mutisya Daniel, Prakash Sandhya, Ranganna Kasturi, Thilagavathi Ramasamy
机构信息
Department of Pharmaceutical Sciences, College of Pharmacy and Health Sciences, Texas Southern University, Houston, TX, USA.
Department of Science and Mathematics, Albany State University, Albany, GA, USA.
出版信息
Chem Biol Drug Des. 2017 Nov;90(5):665-678. doi: 10.1111/cbdd.12993. Epub 2017 May 16.
Abstract
Small interfering RNAs (siRNAs) are one of the valuable tools to investigate the functions of genes and are also used for gene silencing. It has a wide scope in drug discovery through in vivo target validation. siRNA therapeutics are not optimal drug-like molecules due to poor bioavailability and immunogenic and off-target effects. To overcome the challenges associated with siRNA therapeutics, identification of appropriate chemical modifications that improves the stability, specificity and potency of siRNA is essential. This review focuses on the various chemical modifications and their implications in siRNA therapy.
摘要
小干扰RNA（siRNA）是研究基因功能的重要工具之一，也用于基因沉默。它在通过体内靶点验证进行药物发现方面具有广泛应用。由于生物利用度低、免疫原性和脱靶效应，siRNA疗法并非理想的类药物分子。为克服与siRNA疗法相关的挑战，识别能提高siRNA稳定性、特异性和效力的适当化学修饰至关重要。本综述聚焦于各种化学修饰及其在siRNA疗法中的意义。
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