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dc.contributor.authorBiscans, Annabelle
dc.contributor.authorHaraszti, Reka A
dc.contributor.authorEcheverria, Dimas
dc.contributor.authorMiller, Rachael
dc.contributor.authorDidiot, Marie C.
dc.contributor.authorNikan, Mehran
dc.contributor.authorRoux, Loic
dc.contributor.authorAronin, Neil
dc.contributor.authorKhvorova, Anastasia
dc.date2022-08-11T08:10:52.000
dc.date.accessioned2022-08-23T17:22:57Z
dc.date.available2022-08-23T17:22:57Z
dc.date.issued2018-06-06
dc.date.submitted2019-09-18
dc.identifier.citation<p>Mol Ther. 2018 Jun 6;26(6):1520-1528. doi: 10.1016/j.ymthe.2018.03.019. Epub 2018 Apr 4. <a href="https://doi.org/10.1016/j.ymthe.2018.03.019">Link to article on publisher's site</a></p>
dc.identifier.issn1525-0016 (Linking)
dc.identifier.doi10.1016/j.ymthe.2018.03.019
dc.identifier.pmid29699940
dc.identifier.urihttp://hdl.handle.net/20.500.14038/48854
dc.description.abstractSmall extracellular vesicles (sEVs) show promise as natural nano-devices for delivery of therapeutic RNA, but efficient loading of therapeutic RNA remains a challenge. We have recently shown that the attachment of cholesterol to small interfering RNAs (siRNAs) enables efficient and productive loading into sEVs. Here, we systematically explore the ability of lipid conjugates-fatty acids, sterols, and vitamins-to load siRNAs into sEVs and support gene silencing in primary neurons. Hydrophobicity of the conjugated siRNAs defined loading efficiency and the silencing activity of siRNA-sEVs complexes. Vitamin-E-conjugated siRNA supported the best loading into sEVs and productive RNA delivery to neurons.
dc.language.isoen_US
dc.relation<p><a href="http://www.ncbi.nlm.nih.gov/entrez/query.fcgi?db=pubmed&cmd=Retrieve&list_uids=29699940&dopt=Abstract">Link to Article in PubMed</a></p>
dc.relation.urlhttps://doi.org/10.1016/j.ymthe.2018.03.019
dc.rightsCopyright © 2018 The American Society of Gene and Cell Therapy. Under a Creative Commons license, https://creativecommons.org/licenses/by-nc-nd/4.0/, per article landing page at https://doi.org/10.1016/j.ymthe.2018.03.019.
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/
dc.subjectBiochemistry, Biophysics, and Structural Biology
dc.subjectCell and Developmental Biology
dc.subjectGenetics and Genomics
dc.subjectTherapeutics
dc.titleHydrophobicity of Lipid-Conjugated siRNAs Predicts Productive Loading to Small Extracellular Vesicles
dc.typeJournal Article
dc.source.journaltitleMolecular therapy : the journal of the American Society of Gene Therapy
dc.source.volume26
dc.source.issue6
dc.identifier.legacyfulltexthttps://escholarship.umassmed.edu/cgi/viewcontent.cgi?article=1061&amp;context=rti_pubs&amp;unstamped=1
dc.identifier.legacycoverpagehttps://escholarship.umassmed.edu/rti_pubs/62
dc.identifier.contextkey15358663
refterms.dateFOA2022-08-23T17:22:57Z
html.description.abstract<p>Small extracellular vesicles (sEVs) show promise as natural nano-devices for delivery of therapeutic RNA, but efficient loading of therapeutic RNA remains a challenge. We have recently shown that the attachment of cholesterol to small interfering RNAs (siRNAs) enables efficient and productive loading into sEVs. Here, we systematically explore the ability of lipid conjugates-fatty acids, sterols, and vitamins-to load siRNAs into sEVs and support gene silencing in primary neurons. Hydrophobicity of the conjugated siRNAs defined loading efficiency and the silencing activity of siRNA-sEVs complexes. Vitamin-E-conjugated siRNA supported the best loading into sEVs and productive RNA delivery to neurons.</p>
dc.identifier.submissionpathrti_pubs/62
dc.contributor.departmentGraduate School of Biomedical Sciences, Translational Science Program
dc.contributor.departmentDepartment of Medicine
dc.contributor.departmentProgram in Molecular Medicine
dc.contributor.departmentRNA Therapeutics Institute
dc.source.pages1520-1528


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Copyright © 2018 The American Society of Gene and Cell Therapy. Under a Creative Commons license, https://creativecommons.org/licenses/by-nc-nd/4.0/, per article landing page at https://doi.org/10.1016/j.ymthe.2018.03.019.
Except where otherwise noted, this item's license is described as Copyright © 2018 The American Society of Gene and Cell Therapy. Under a Creative Commons license, https://creativecommons.org/licenses/by-nc-nd/4.0/, per article landing page at https://doi.org/10.1016/j.ymthe.2018.03.019.