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dc.contributor.authorBaigude, Huricha
dc.contributor.authorSu, Jie
dc.contributor.authorMcCarroll, Joshua
dc.contributor.authorRana, Tariq M.
dc.date2022-08-11T08:08:29.000
dc.date.accessioned2022-08-23T15:57:12Z
dc.date.available2022-08-23T15:57:12Z
dc.date.issued2013-08-08
dc.date.submitted2014-05-13
dc.identifier.citation<p>Baigude H, Su J, McCarroll J, Rana TM. In Vivo Delivery of RNAi by Reducible Interfering Nanoparticles (iNOPs). ACS Med Chem Lett. 2013 Aug 8;4(8):720-723. <a href="http://dx.doi.org/10.1021/ml4001003">Link to article on publisher's site</a></p>
dc.identifier.issn1948-5875 (Linking)
dc.identifier.doi10.1021/ml4001003
dc.identifier.pmid24319542
dc.identifier.urihttp://hdl.handle.net/20.500.14038/30141
dc.description.abstractRNA interference (RNAi) has considerable potential as a therapeutic strategy, but the development of efficient in vivo RNA delivery methods remains challenging. To this end, we designed and synthesized chemically modified interfering nanoparticles (iNOPs) composed of functionalized poly-l-lysine dendrimers modified with reducible spacers to facilitate release of small interfering RNAs (siRNAs) in vivo. We show that the novel siRNA-iNOP complexes mediate efficient gene-specific RNAi in cultured cells and in mice, where they display enhanced tissue-targeting capabilities. At a clinically feasible dose of 1 mg kg-1, apolipoprotein B (apoB) siRNA-iNOP complexes achieved approximately 40-45% reduction of liver apoB mRNA and plasma apoB protein levels within 48 h of administration to mice, without apparent toxicity. Collectively, these findings demonstrate that siRNA delivery by the modified reducible iNOPs can provide a clinically significant and potentially tissue-specific new approach for RNAi therapy.
dc.language.isoen_US
dc.relation<p><a href="http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&list_uids=24319542&dopt=Abstract">Link to Article in PubMed</a></p>
dc.relation.urlhttp://www.ncbi.nlm.nih.gov/pmc/articles/PMC3850243/
dc.subjectNanoparticle
dc.subjectiNOP
dc.subjectreducible nanoparticles
dc.subjectsiRNA delivery
dc.subjectAnimal Experimentation and Research
dc.subjectBiochemistry
dc.subjectCells
dc.subjectGenetic Phenomena
dc.subjectInvestigative Techniques
dc.subjectMedicinal and Pharmaceutical Chemistry
dc.subjectMedicinal-Pharmaceutical Chemistry
dc.subjectMolecular Genetics
dc.subjectNucleic Acids, Nucleotides, and Nucleosides
dc.subjectTherapeutics
dc.titleIn Vivo Delivery of RNAi by Reducible Interfering Nanoparticles (iNOPs)
dc.typeJournal Article
dc.source.journaltitleACS medicinal chemistry letters
dc.source.volume4
dc.source.issue8
dc.identifier.legacycoverpagehttps://escholarship.umassmed.edu/faculty_pubs/381
dc.identifier.contextkey5574360
html.description.abstract<p>RNA interference (RNAi) has considerable potential as a therapeutic strategy, but the development of efficient in vivo RNA delivery methods remains challenging. To this end, we designed and synthesized chemically modified interfering nanoparticles (iNOPs) composed of functionalized poly-l-lysine dendrimers modified with reducible spacers to facilitate release of small interfering RNAs (siRNAs) in vivo. We show that the novel siRNA-iNOP complexes mediate efficient gene-specific RNAi in cultured cells and in mice, where they display enhanced tissue-targeting capabilities. At a clinically feasible dose of 1 mg kg-1, apolipoprotein B (apoB) siRNA-iNOP complexes achieved approximately 40-45% reduction of liver apoB mRNA and plasma apoB protein levels within 48 h of administration to mice, without apparent toxicity. Collectively, these findings demonstrate that siRNA delivery by the modified reducible iNOPs can provide a clinically significant and potentially tissue-specific new approach for RNAi therapy.</p>
dc.identifier.submissionpathfaculty_pubs/381
dc.contributor.departmentDepartment of Biochemistry and Molecular Pharmacology
dc.source.pages720-723


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