Enhance transgene responses through improving cellular uptake and intracellular trafficking by bio-inspired non-viral vectors
Enhance transgene responses through improving cellular uptake and intracellular trafficking by bio-inspired non-viral vectors
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通过仿生非病毒载体改善细胞摄取和细胞内运输，增强转基因反应
DOI：
10.1186/s12951-020-0582-z
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发表时间：
2020-01
影响因子：
10.2
通讯作者：
Zhang Bang-Le
中科院分区：
文献类型：
--
作者：
Ma Xi-Xi;Xu Jing-Liang;Jia Yi-Yang;Zhang Ya-Xuan;Wang Wei;Li Chen;He Wei;Zhou Si-Yuan;Zhang Bang-Le
BackgroundGene therapy remains a significant challenge due to lots of barriers limiting the genetic manipulation technologies. As for non-viral delivery vectors, they often suffer insufficient performance due to inadequate cellular uptake and gene degradation in endosome or lysosome. The importance of overcoming these conserved intracellular barriers is increasing as the delivery of genetic cargo.ResultsA surface-functionalized non-viral vector involving the biomimetic mannitol moiety is initiated, which can control the cellular uptake and promote the caveolae-mediated pathway and intracellular trafficking, thus avoiding acidic and enzymatic lysosomal degradation of loaded gene internalized by clathrin-mediated pathway. Different degrees of mannitol moiety are anchored onto the surface of the nanoparticles to form bio-inspired non-viral vectors and CaP-MA-40 exhibits remarkably high stability, negligible toxicity, and significantly enhanced transgene expression both in vitro and in vivo.ConclusionsThis strategy highlights a paradigmatic approach to construct vectors that need precise intracellular delivery for innovative applications.
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影响因子：
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作者：
T. Akiyama;H. Ogawara
通讯作者：
T. Akiyama;H. Ogawara
影响因子：
5.3
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影响因子：
10.9
作者：
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L. Pasquet;E. Bellard;S. Chabot;B. Markelc;M. Rols;J. Teissié;M. Golzio
影响因子：
4.9
作者：
Reilly, Meghan J.;Larsen, John D.;Sullivan, Millicent O.
通讯作者：
Sullivan, Millicent O.
影响因子：
16.1
作者：
R. Wattiaux;Nathanael Laurent;S. Coninck;M. Jadot
通讯作者：
R. Wattiaux;Nathanael Laurent;S. Coninck;M. Jadot