Strategies for in vivo targeted gene silencing

Strategies for in vivo targeted gene silencing
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体内靶向基因沉默策略

DOI:
10.5372/abm.v7i4.31738
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发表时间:
2013-08
期刊:
影响因子:
0.6
通讯作者:
Lianfang Du
Lianfang Du
中科院分区:
医学4区
文献类型:
--
作者:
Hanghui Wang;Yixin Song;Lianfang Du

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背景资料:小干扰RNA(small interfering RNA,siRNA)在人类疾病的研究、诊断和治疗中具有重要的应用前景。然而,siRNA在体内特异性和有效地递送到细胞中仍然是一个巨大的挑战。siRNA的靶向修饰、基于病毒纳米颗粒的载体、靶向多功能/多级纳米系统、超声靶向微泡破坏和肿瘤靶向在一体化系统中提供了有用的多模式靶向递送方法。目的:我们提供了siRNA递送的不同策略的概述,包括siRNA、纳米颗粒和病毒载体的直接修饰。方法:检索标准数据库。相关的主要和摘要资源被确定和提取。结果:总结了体内靶向基因沉默的策略,包括靶细胞,靶基因,靶传说,和疾病模型的每一个策略。综述了siRNA递送的策略,旨在在体内靶向基因沉默。结论:将病毒或非病毒载体的优势整合到基因沉默中可能会对生物医学研究产生深远的影响。最近的进展正在寻找答案。关键词:基因递送,微泡,纳米颗粒,RNA干扰,靶向,超声,载体
Background: Small interfering RNA (siRNA) has attracted extensive attention showing significant promise for the study, diagnosis, and treatment of human disease. However, the specific and efficient delivery of siRNA into cells in vivo remains a great challenge. Targeted modification of siRNA, viral nanoparticle-based vectors, targeted multifunctional/multistage nanosystems, combining ultrasound-targeted microbubble destruction, and tumor targeting in an all-in-one system, provides a useful multimodal approach in targeted delivery.  Objective: We provided an overview of different strategies for siRNA delivery including direct modification of siRNA, nanoparticles and viral vectors. Methods: We conducted a search of standard database. Relevant primary and summary resources were identified and abstracted. A summary of strategies for in vivo targeted gene silencing was produced.  Results: A list of strategies for gene-targeted delivery in vivo was summarized, including target cells, target genes, target legends, and disease model for each strategy. An overview of strategies for siRNA delivery aimed at in vivo targeted gene silencing was presented. Conclusions: Integration of the advantages of viral or nonviral vectors into gene silencing could have profound impacts on biomedical research. Recent progress is pointing at answers. Keywords: Gene delivery, microbubbles, nanoparticles, RNA interference, targeting, ultrasound, vector
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