小干扰RNA
RNA干扰
基因沉默
核糖核酸
计算生物学
小RNA
信使核糖核酸
精密医学
生物信息学
RNA沉默
医学
生物
遗传增强
个性化医疗
基因表达
基因
转化研究
免疫系统
翻译(生物学)
反式siRNA
作者
Ritu Dahiya,Ajay Pal Singh,Pooja Mathur,Laxmi Rani,Ebrahim Ahmed Jaber Al Hatef,Hadeel Khalid Alharbi,Parakh Basist,Sradhanjali Mohapatra,Kanika Goyal
标识
DOI:10.2174/0115665232434772251211065217
摘要
Small interfering RNA (siRNA) and messenger RNA (mRNA) therapeutics represent transformative approaches in precision pharmacology, enabling targeted gene silencing and protein expression, respectively. This review provides a comprehensive analysis of their molecular mechanisms, delivery strategies, clinical applications, and future directions. By leveraging RNA interference for siRNA and translational machinery for mRNA, these therapeutics address previously untreatable diseases, including genetic disorders, cancers, and infectious diseases. Advances in nanoparticle- based delivery systems, such as lipid nanoparticles, have overcome historical barriers like RNA instability and immune activation. Current clinical applications, including FDA-approved therapies, highlight their efficacy, while ongoing challenges, such as off-target effects and scalable production, are discussed. Future directions emphasize personalized medicine, combination therapies, and novel delivery platforms to enhance therapeutic precision and accessibility.
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