Engineering RNA for targeted siRNA delivery and medical application.
Engineering RNA for targeted siRNA delivery and medical application.
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DOI：
10.1016/j.addr.2010.03.008
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发表时间：
2010-04-30
影响因子：
16.1
通讯作者：
Shu, Yi
中科院分区：
文献类型：
--
作者：
Guo, Peixuan;Coban, Oana;Snead, Nicholas M.;Trebley, Joe;Hoeprich, Steve;Guo, Songchuan;Shu, Yi
关键词：
用于纳米技术和医学应用的RNA工程是一个令人兴奋的新兴研究领域。RNA在纳米尺度上具有内在定义的特征，并且由于其结构和功能的惊人多样性，灵活性和多功能性而成为此类应用的特别有趣的候选者。具体来说，目前使用siRNA沉默与疾病有关的靶基因在科学界引起了很大的兴奋。以时间和空间控制的方式序列特异性下调基因表达的内在能力已经引起了对基于siRNA的疗法的高度关注和快速发展。尽管用于基因沉默的方法已经在体外以高效率和特异性实现，但是核酸在体内有效递送至特定细胞一直是RNA治疗的障碍。本文介绍了用于诊断、预防和治疗人类疾病的各种基于RNA的方法，重点介绍了用于体内递送的siRNA的非病毒载体的最新发展。siRNA治疗的应用和挑战，以及这些问题的潜在解决方案，使用phi29 pRNA为基础的载体作为多价载体特异性递送siRNA，核酶，药物或其他治疗剂到特定细胞的治疗方法也将得到解决。
RNA engineering for nanotechnology and medical applications is an exciting emerging research field. RNA has intrinsically defined features on the nanometre scale and is a particularly interesting candidate for such applications due to its amazing diversity, flexibility and versatility in structure and function. Specifically, the current use of siRNA to silence target genes involved in disease has generated much excitement in the scientific community. The intrinsic ability to sequence-specifically downregulate gene expression in a temporally- and spatially controlled fashion has led to heightened interest and rapid development of siRNA-based therapeutics. Although methods for gene silencing have been achieved with high efficacy and specificity in vitro, the effective delivery of nucleic acids to specific cells in vivo has been a hurdle for RNA therapeutics. This article covers different RNA-based approaches for diagnosis, prevention and treatment of human disease, with a focus on the latest developments of non-viral carriers of siRNA for delivery in vivo. The applications and challenges of siRNA therapy, as well as potential solutions to these problems, the approaches for using phi29 pRNA-based vectors as polyvalent vehicles for specific delivery of siRNA, ribozymes, drugs or other therapeutic agents to specific cells for therapy will also be addressed.
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影响因子：
6.2
作者：
Ai, Zhihong;Yin, Lianhua;Feng, Youji
通讯作者：
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DOI：
10.1038/nrd2310
发表时间：
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期刊：
Nature reviews. Drug discovery
影响因子：
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作者：
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适体介导的siRNA传递。
影响因子：
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作者：
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影响因子：
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作者：
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通讯作者：
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影响因子：
2.2
作者：
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通讯作者：
Akkina R