A highly efficient synthetic vector: nonhydrodynamic delivery of DNA to hepatocyte nuclei in vivo.
A highly efficient synthetic vector: nonhydrodynamic delivery of DNA to hepatocyte nuclei in vivo.
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DOI：
10.1021/nn4012384
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发表时间：
2013-06-25
期刊：
影响因子：
17.1
通讯作者：
Huang, Leaf
中科院分区：
文献类型：
--
作者：
Hu, Yunxia;Haynes, Matthew T.;Wang, Yuhua;Liu, Feng;Huang, Leaf
由磷酸钙核、富含精氨酸的多肽、阳离子和聚乙二醇化的脂膜以及半乳糖靶向配体组成的多功能膜核纳米颗粒已被开发为有效的核传递载体和随后在体内肝细胞中的基因表达的合成载体。靶向颗粒表现出快速和广泛的肝脏蓄积，并主要被肝细胞内化，而LCP中包含这样的多肽足以引发高度的质粒DNA核转位。单环CR8C显著增强体内基因的表达，是线性CR8C的十倍以上，这可能是由于DNA/肽复合体的释放促进机制。尽管这种制剂的活性比流体动力注射的活性低100倍，但它是一种侵入性小得多的替代品。据我们所知，这是最有效的肝脏基因转移的合成载体。
Multifunctional membrane-core nanoparticles, composed of calcium phosphate cores, arginine-rich peptides, cationic and PEGylated lipid membranes, and galactose targeting ligands, have been developed as synthetic vectors for efficient nuclear delivery of plasmid DNA and subsequent gene expression in hepatocytes in vivo. Targeted particles exhibited rapid and extensive hepatic accumulation and were predominantly internalized by hepatocytes, while the inclusion of such peptides in LCP was sufficient to elicit high degrees of nuclear translocation of plasmid DNA. Monocyclic CR8C significantly enhanced in vivo gene expression over ten-fold more than linear CR8C, likely due to a release-favoring mechanism of the DNA/peptide complex. Though 100-fold lower in activity than that achieved via hydrodynamic injection, this formulation presents as a much less invasive alternative. To our knowledge, this is the most effective synthetic vector for liver gene transfer.
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影响因子：
17.1
作者：
Ashley CE;Carnes EC;Epler KE;Padilla DP;Phillips GK;Castillo RE;Wilkinson DC;Wilkinson BS;Burgard CA;Kalinich RM;Townson JL;Chackerian B;Willman CL;Peabody DS;Wharton W;Brinker CJ
通讯作者：
Brinker CJ
影响因子：
5.1
作者：
Khalil, I. A.;Kogure, K.;Harashima, H.
通讯作者：
Harashima, H.
影响因子：
2.9
作者：
Douglas, Kimberly L.
通讯作者：
Douglas, Kimberly L.
用于基因传递的精氨酸-壳聚糖/DNA 自组装纳米颗粒：体外特性和转染效率。
影响因子：
5.8
作者：
Gao, Yu;Xu, Zhenghong;Li, Yaping
通讯作者：
Li, Yaping
影响因子：
5.1
作者：
Liu, F;Song, YK;Liu, D
通讯作者：
Liu, D