title: Dendrimer Nanovectors for SiRNA Delivery.
abstract: Small interfering RNA (SiRNA) delivery remains a major challenge in RNAi-based therapy. Dendrimers are emerging as appealing nonviral vectors for SiRNA delivery thanks to their well-defined architecture and their unique cooperativity and multivalency confined within a nanostructure. We have recently demonstrated that structurally flexible poly(amidoamine) (PAMAM) dendrimers are safe and effective nanovectors for SiRNA delivery in various disease models in vitro and in vivo. The present chapter showcases these dendrimers can package different SiRNA molecules into stable and nanosized particles, which protect SiRNA from degradation and promote cellular uptake of SiRNA, resulting in potent gene silencing at both mRNA and protein level in the prostate cancer cell model. Our results demonstrate this set of flexible PAMAM dendrimers are indeed safe and effective nonviral vectors for SiRNA delivery and hold great promise for further applications in functional genomics and RNAi-based therapies.
authors.affiliation: Aix-Marseille Université, CNRS CINaM UMR 7325, 163 avenue de Luminy, 13288, Marseille Cedex 09, France.
authors.forename: Xiaoxuan
authors.initials: X
authors.lastname: Liu
authors.affiliation: Aix-Marseille Université, CNRS CINaM UMR 7325, 163 avenue de Luminy, 13288, Marseille Cedex 09, France. ling.peng@univ-amu.fr.
authors.forename: Ling
authors.initials: L
authors.lastname: Peng
mesh_terms: D001692:Biological Transport; D045744:Cell Line, Tumor; D050091:Dendrimers / Q000737:chemistry* / Q000378:metabolism; D004337:Drug Carriers / Q000737:chemistry* / Q000378:metabolism; D004355:Drug Stability; D005786:Gene Expression Regulation / Q000235:genetics; D020868:Gene Silencing; D055551:HSP27 Heat-Shock Proteins / Q000172:deficiency / Q000235:genetics; D006801:Humans; D018613:Microscopy, Confocal; D053758:Nanoparticles / Q000737:chemistry*; D012333:RNA, Messenger / Q000235:genetics / Q000378:metabolism; D034741:RNA, Small Interfering / Q000737:chemistry* / Q000235:genetics; D012259:Ribonuclease, Pancreatic / Q000378:metabolism
chemical_list: D050091:Dendrimers; D004337:Drug Carriers; D055551:HSP27 Heat-Shock Proteins; C104700:PAMAM Starburst; D012333:RNA, Messenger; D034741:RNA, Small Interfering; D012259:Ribonuclease, Pancreatic
keywords: Dendrimer; Nonviral vectors; RNA interference; SiRNA delivery