Development of Excipient-Free Freeze-Dryable Unimolecular Hyperstar Polymers for Efficient siRNA Silencing.
Development of Excipient-Free Freeze-Dryable Unimolecular Hyperstar Polymers for Efficient siRNA Silencing.
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DOI：
10.1021/acsmacrolett.7b00242
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发表时间：
2017-06
影响因子：
5.8
通讯作者：
Liang Zhao;Xiang Wu;Xiaofeng Wang;Chunying Duan;Hongyan Wang;Amol D. Punjabi;Yang Zhao;Yuanwei Zhang;Zuoshang Xu;Haifeng Gao;Gang Han
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文献类型：
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作者：
Liang Zhao;Xiang Wu;Xiaofeng Wang;Chunying Duan;Hongyan Wang;Amol D. Punjabi;Yang Zhao;Yuanwei Zhang;Zuoshang Xu;Haifeng Gao;Gang Han
我们设计了一种单分子超星形聚合物，用于有效的小干扰RNA（siRNA）递送，其可以在反复冻干和重构下加工，而不需要任何赋形剂。超星形聚合物含有可生物降解的超支化核心，并通过从其核心辐射出的数千个阳离子臂与siRNA结合。siRNA/hyperstar复合物显示siRNA转染效率上级Lipofectamine 2000的人Cu，Zn超氧化物歧化酶1（SOD 1）基因，其突变导致家族性肌萎缩侧索硬化症。更重要的是，超星聚合物作为单分子容器最大限度地减少了冻干过程中的多聚物交叉相互作用，并且这在重复冷冻干燥和重构后保持了siRNA/超星复合物独特的高转染效率，而无需常规的赋形剂。
We designed a unimolecular hyperstar polymer for efficient small interfering RNA (siRNA) delivery that can be processed under repeated lyophilization and reconstitution without the need of any excipient. The hyperstar polymer contains a biodegradable hyperbranched core and is bound to siRNA through its thousands of cationic arms that radiate from its core. The siRNA/hyperstar complexes showed siRNA transfection efficiency that was superior to that of Lipofectamine2000 in regard to the gene for human Cu, Zn superoxide dismutase 1 (SOD1), whose mutation causes familial amyotrophic lateral sclerosis. More importantly, hyperstar polymers as unimolecular containers minimized the multipolymer cross-interaction during lyophilization, and this maintained the uniquely high transfection efficiency of the siRNA/hyperstar complexes after repeated freeze-drying and reconstitution without the conventional need for excipient.