Dana Hassan, Chalbatani Ghanbar Mahmoodi, Mahmoodzadeh Habibollah, Karimloo Rezvan, Rezaiean Omid, Moradzadeh Amirreza, Mehmandoost Narges, Moazzen Fateme, Mazraeh Ali, Marmari Vahid, Ebrahimi Mohammad, Rashno Mohammad Menati, Abadi Saeid Jan, Gharagouzlo Elahe
Department of Biology, Damghan Branch, Islamic Azad University, Damghan, Iran.
Cancer Research Center, Cancer Institute of Iran, Tehran University of Medical Science, Tehran, Iran.
Int J Biomed Sci. 2017 Jun;13(2):48-57.
One of the most important advances in biology has been the discovery that siRNA (small interfering RNA) is able to regulate the expression of genes, by a phenomenon known as RNAi (RNA interference). The discovery of RNAi, first in plants and and later in mammalian cells, led to the emergence of a transformative view in biomedical research. siRNA has gained attention as a potential therapeutic reagent due to its ability to inhibit specific genes in many genetic diseases. siRNAs can be used as tools to study single gene function both in vivo and in-vitro and are an attractive new class of therapeutics, especially against undruggable targets for the treatment of cancer and other diseases. The siRNA delivery systems are categorized as non-viral and viral delivery systems. The non-viral delivery system includes polymers; Lipids; peptides etc. are the widely studied delivery systems for siRNA. Effective pharmacological use of siRNA requires 'carriers' that can deliver the siRNA to its intended site of action. The carriers assemble the siRNA into supramolecular complexes that display functional properties during the delivery process.
生物学领域最重要的进展之一是发现小干扰RNA（siRNA）能够通过一种称为RNA干扰（RNAi）的现象来调节基因表达。RNAi首先在植物中被发现，随后在哺乳动物细胞中被发现，这一发现引发了生物医学研究中的一种变革性观点。由于siRNA能够在许多遗传疾病中抑制特定基因，它作为一种潜在的治疗试剂受到了关注。siRNA可作为工具在体内和体外研究单个基因的功能，并且是一类有吸引力的新型治疗药物，特别是针对癌症和其他疾病中难以成药的靶点。siRNA递送系统可分为非病毒递送系统和病毒递送系统。非病毒递送系统包括聚合物、脂质、肽等，是广泛研究的siRNA递送系统。siRNA的有效药理学应用需要“载体”将siRNA递送至其预期的作用位点。这些载体将siRNA组装成超分子复合物，在递送过程中表现出功能特性。
