Key Laboratory of Polymer Ecomaterials, Changchun Institute of Applied Chemistry, Chinese Academy of Sciences , Changchun, China.
Mol Ther Methods Clin Dev. 2016 Apr 6;3:16023. doi: 10.1038/mtm.2016.23. eCollection 2016.
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.
基因治疗是一种有前途的策略，通过有意改变病理细胞中的基因表达，可以特异性治疗许多与基因相关的人类疾病。基因治疗的成功临床应用取决于高效的基因传递系统。许多人已经尝试提高基因治疗的安全性和效率。由于纳米颗粒的尺寸、形状、表面和生物行为可调，因此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传递的纳米颗粒的临床发展。将简要回顾几种最接近临床应用的有前途的候选物。然后，将确定并总结用于临床基因治疗的纳米颗粒的最新进展。最后，对未来临床基因传递用纳米颗粒的发展进行了展望。
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