RNA Therapeutics Institute; Program in Molecular Medicine; Department of Medicine; Graduate School of Biomedical Sciences, Translational Science Program
Biochemistry, Biophysics, and Structural Biology | Cell and Developmental Biology | Genetics and Genomics | Therapeutics
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.
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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.
DOI of Published Version
Mol Ther. 2018 Jun 6;26(6):1520-1528. doi: 10.1016/j.ymthe.2018.03.019. Epub 2018 Apr 4. Link to article on publisher's site
Molecular therapy : the journal of the American Society of Gene Therapy
Biscans A, Haraszti RA, Echeverria D, Miller R, Didiot MC, Nikan M, Roux L, Aronin N, Khvorova A. (2018). Hydrophobicity of Lipid-Conjugated siRNAs Predicts Productive Loading to Small Extracellular Vesicles. RNA Therapeutics Institute Publications. https://doi.org/10.1016/j.ymthe.2018.03.019. Retrieved from https://escholarship.umassmed.edu/rti_pubs/62
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This work is licensed under a Creative Commons Attribution-Noncommercial-No Derivative Works 4.0 License.