siRNA-based therapeutic approaches for rheumatic diseases
siRNA-based therapeutic approaches for rheumatic diseases
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DOI：
10.1038/nrrheum.2012.176
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发表时间：
2013-01-01
影响因子：
33.7
通讯作者：
Jorgensen, Christian
中科院分区：
文献类型：
--
作者：
Apparailly, Florence;Jorgensen, Christian
RNA干扰（RNAi）是过去几十年来最令人兴奋和最重要的发现之一。小干扰RNA（siRNA）可以沉默基因活性，并用于干扰病理生理过程。大量的研究集中在将“药物样”特性--稳定性、选择性和效力--引入RNAi分子，并且已经开始了临床试验。尽管取得了初步成功，但目前的挑战仍然是开发优化的载体，避免脱靶效应，同时有效地将治疗性siRNA递送到特定的细胞类型。对于许多其他疾病，基于siRNA的治疗正在成为治疗风湿性疾病的一种有前途的方法。虽然风湿性疾病的发病机制是复杂的，但能够影响炎症或结构损伤的候选基因的鉴定已经成功。在这里，我们将讨论该领域的进展和潜在的应用siRNA疗法在风湿性疾病的临床试验。显然，F. & Jorgensen，C.国家风湿病学会9，56-62（2013）; 2012年10月23日在线发表; doi：10.1038/nrrheum.2012.176
RNA interference (RNAi) is one of the most exciting and important discoveries of the past few decades. Small interfering RNAs (siRNAs) can silence gene activity and be used to interfere with pathophysiological processes. Substantial research has focused on introducing 'drug-like' properties-stability, selectivity and potency-to RNAi molecules, and clinical trials have been initiated. Despite initial success, the current challenge that remains is to develop optimized vehicles that avoid off-target effects whilst efficiently delivering the therapeutic siRNA to specific cell types. As for many other diseases, siRNA-based therapy is emerging as a promising approach for the treatment of rheumatic disorders. Although the pathogenesis of rheumatic diseases is complex, identification of candidate genes able to influence either inflammation or structural damage has been successful. Here, we will discuss advances in the field and potential applications of siRNA therapeutics in clinical trials for rheumatic conditions. Apparailly, F. & Jorgensen, C. Nat. Rev. Rheumatol. 9, 56-62 (2013); published online 23 October 2012; doi:10.1038/nrrheum.2012.176