Production and clinical development of nanoparticles for gene delivery.
Production and clinical development of nanoparticles for gene delivery.
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用于基因递送的纳米粒子的生产和临床开发
DOI：
10.1038/mtm.2016.23
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发表时间：
2016
期刊：
影响因子：
--
通讯作者：
Chen X
中科院分区：
文献类型：
--
作者：
Chen J;Guo Z;Tian H;Chen X
基因治疗是一种有希望的策略，通过故意改变病理细胞中的基因表达来特异性治疗许多基因相关的人类疾病。基因治疗的成功应用依赖于高效的基因传递系统。人们已经尝试了许多努力来提高基于基因的疗法的安全性和有效性。纳米粒子由于其尺寸、形状、表面和生物学行为的可调性，已被证明是最有前途的临床基因治疗载体。在这篇综述中，将特别强调用于基因递送的纳米颗粒的临床开发。几个有前途的候选人，这是最接近临床应用，将简要审查。最后，对纳米粒子在临床基因治疗中的最新进展进行了归纳和总结。最后对纳米粒在临床基因治疗中的应用前景进行了展望。
Gene therapy is a promising strategy for specific treatment of numerous gene-associated human diseases by intentionally altering the gene expression in pathological cells. A successful clinical application of gene-based therapy depends on an efficient gene delivery system. Many efforts have been attempted to improve the safety and efficiency of gene-based therapies. Nanoparticles have been proved to be the most promising vehicles for clinical gene therapy due to their tunable size, shape, surface, and biological behaviors. In this review, the clinical development of nanoparticles for gene delivery will be particularly highlighted. Several promising candidates, which are closest to clinical applications, will be briefly reviewed. Then, the recent developments of nanoparticles for clinical gene therapy will be identified and summarized. Finally, the development of nanoparticles for clinical gene delivery in future will be prospected.