Amphiphilic Dendrimer Vectors for RNA Delivery:State-of-the-Art and Future

作     者：Jiaxuan Chen Dandan Zhu Xiaoxuan Liu Ling Peng 

作者机构：Aix Marseille UniversitéCNRSCenter Interdisciplinaire de Nanoscience de MarseilleCINaM UMR 7325“Equipe Labellisée Ligue Contre le Cancer”13288 MarseilleFrance State Key Laboratory of Natural Medicines and Jiangsu Key Laboratory of Drug Discovery for Metabolic DiseasesCenter of Drug DiscoveryCenter of Advanced Pharmaceuticals and BiomaterialsChina Pharmaceutical University210009 NanjingP.R.China 

出 版 物：《Accounts of Materials Research》 (材料研究述评（英文）)

年 卷 期：2022年第3卷第5期

页      面：484-497页

学科分类：081704[工学-应用化学] 07[理学] 08[工学] 0817[工学-化学工程与技术] 070305[理学-高分子化学与物理] 080501[工学-材料物理与化学] 0805[工学-材料科学与工程（可授工学、理学学位）] 0703[理学-化学] 

基　　金：We are grateful for financial support from the Ligue Nationale Contre le Cancer(L.P.),Campus France PHC program CAI YUANPEI(L.P.,X.L) the French National Research Agency for funding of the Era-Net EURONANOMED European Research projects"Target4Cancer",“NANOGLIO",“INANO-GUN"(LP.) the European Union's Horizon 2020 research and innovation program H2020 NMBP“SAFE-N-MED-TECH”(No.814607,LP.,XL.) the China Schol arship Council(.C.),Campus France bourse Eiffel(.C.),CNRS Aix-Marseille Universite 

主　　题：breakthrough retaining desired 

摘      要：Dendrimers,a special family of polymers,are particularly promising materials for various biomedical applications by virtue of their well-defined dendritic structure and cooperative ***,in this Account,we present state-of-the-art amphiphilic dendrimers for nucleic acid *** acid(RNA)molecules are fast becoming an important drug modality,particularly since the recent success of mRNA vaccines against ***,RNA therapeutics offer the unique opportunity to treat diseases at the gene level and address“undruggable***,RNA therapeutics are not stable and have poor bioavailability,imposing the need for their protection and safe delivery by vectors to the sites-of-action to allow the desired therapeutic ***,the two most advanced nonviral vectors are based on lipids and polymers,with lipid vectors primarily exploiting the membrane-fusion mechanism and polymer vectors mainly endocytosis-mediated ***,only lipid vectors have been advanced through to their clinical use in the delivery of,for example,the first siRNA drug and the first mRNA *** success of lipid vectors for RNA delivery has motivated research for further innovative materials as delivery ***,we have pioneered lipid/dendrimer conjugates,referred to as amphiphilic dendrimers,for siRNA delivery with the view to harnessing the delivery advantages of both lipid and polymer vectors while enjoying the unique structural features of *** amphiphilic dendrimer vectors are lipid/dendrimer hybrids and are thus able to mimic lipid vectors and exploit membranefusion-mediated delivery,while simultaneously retaining the multivalent properties of polymer vectors that allow endocytosis-based *** addition,they have precisely controllable and stable nanosized chemical structures and offer nanotechnology-based *** amphiphilic dendrimer vectors share two important elements:chemical hydrophilic