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dc.contributor.authorMizrak, Arda
dc.contributor.authorBolukbasi, Mehmet Fatih
dc.contributor.authorOzdener, Gokhan Baris
dc.contributor.authorBrenner, Gary Jay
dc.contributor.authorMadlener, Sibylle
dc.contributor.authorErkan, Erdogan Pekcan
dc.contributor.authorStröbel, Thomas
dc.contributor.authorBreakefield, Xandra Owens
dc.contributor.authorSaydam, Okay
dc.date.accessioned2013-05-05T00:35:41Z
dc.date.issued2013
dc.identifier.citationMizrak, Arda, Mehmet Fatih Bolukbasi, Gokhan Baris Ozdener, Gary J. Brenner, Sibylle Madlener, Erdogan Pekcan Erkan, Thomas Ströbel, Xandra O. Breakefield, and Okay Saydam. 2013. Genetically engineered microvesicles carrying suicide mRNA/protein inhibit schwannoma tumor growth. Molecular Therapy 21(1): 101-108.en_US
dc.identifier.issn1525-0016en_US
dc.identifier.issn1525-0024en_US
dc.identifier.urihttp://nrs.harvard.edu/urn-3:HUL.InstRepos:10610865
dc.description.abstractMicrovesicles (MVs) play an important role in intercellular communication by carrying mRNAs, microRNAs (miRNAs), non-coding RNAs, proteins, and DNA from cell to cell. To our knowledge, this is the first report of delivery of a therapeutic mRNA/protein via MVs for treatment of cancer. We first generated genetically engineered MVs by expressing high levels of the suicide gene mRNA and protein–cytosine deaminase (CD) fused to uracil phosphoribosyltransferase (UPRT) in MV donor cells. MVs were isolated from these cells and used to treat pre-established nerve sheath tumors (schwannomas) in an orthotopic mouse model. We demonstrated that MV-mediated delivery of CD-UPRT mRNA/protein by direct injection into schwannomas led to regression of these tumors upon systemic treatment with the prodrug (5-fluorocytosine (5-FC)), which is converted within tumor cells to 5-fluorouracil (5-FU)–an anticancer agent. Taken together, these studies suggest that MVs can serve as novel cell-derived “liposomes” to effectively deliver therapeutic mRNA/proteins to treatment of diseases.en_US
dc.language.isoen_USen_US
dc.publisherNature Publishing Groupen_US
dc.relation.isversionofdoi:10.1038/mt.2012.161en_US
dc.relation.hasversionhttp://www.ncbi.nlm.nih.gov/pmc/articles/PMC3538300/pdf/en_US
dash.licenseLAA
dc.titleGenetically Engineered Microvesicles Carrying Suicide mRNA/Protein Inhibit Schwannoma Tumor Growthen_US
dc.typeJournal Articleen_US
dc.description.versionVersion of Recorden_US
dc.relation.journalMolecular Therapyen_US
dash.depositing.authorBrenner, Gary Jay
dc.date.available2013-05-05T00:35:41Z
dc.identifier.doi10.1038/mt.2012.161*
dash.contributor.affiliatedBrenner, Gary
dash.contributor.affiliatedBreakefield, Xandra


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