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反义抑制剂、核酶和小干扰RNA。

Antisense inhibitors, ribozymes, and siRNAs.

作者信息

Thompson Alexander J V, Patel Keyur

机构信息

Division of Gastroenterology/Hepatology, Duke Clinical Research Institute, Duke University, Durham, NC 27715, USA.

出版信息

Clin Liver Dis. 2009 Aug;13(3):375-90. doi: 10.1016/j.cld.2009.05.003.

Abstract

The current standard of care for the treatment of hepatitis C virus infection, pegylated interferon-alpha and ribavirin, is costly, associated with significant side effects, and effective in only 50% of patients. There is therefore a need for the development of novel antiviral therapies. One such approach involves the application of gene silencing technologies, including antisense oligonucleotides, ribozymes, RNA interference, and aptamers. However, despite great scientific advances over the past decade, and promising in vitro data, several significant challenges continue to limit the translation of this technology to the clinical setting. This review provides a concise update of the current literature.

摘要

目前治疗丙型肝炎病毒感染的标准疗法——聚乙二醇化干扰素-α和利巴韦林,成本高昂,伴有显著副作用,且仅对50%的患者有效。因此,需要开发新型抗病毒疗法。一种方法是应用基因沉默技术,包括反义寡核苷酸、核酶、RNA干扰和适配体。然而,尽管在过去十年取得了巨大的科学进展,且体外实验数据很有前景,但仍有几个重大挑战继续限制着这项技术向临床应用的转化。本综述简要介绍了当前的文献。

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