RNA Therapeutics Institute Publications
UMMS Affiliation
RNA Therapeutics Institute; Program in Molecular Medicine; Department of Medicine; Graduate School of Biomedical Sciences, Translational Science Program
Publication Date
2018-06-06
Document Type
Article
Disciplines
Biochemistry, Biophysics, and Structural Biology | Cell and Developmental Biology | Genetics and Genomics | Therapeutics
Abstract
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.
Rights and Permissions
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
10.1016/j.ymthe.2018.03.019
Source
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
Journal/Book/Conference Title
Molecular therapy : the journal of the American Society of Gene Therapy
Related Resources
PubMed ID
29699940
Repository Citation
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
Creative Commons License
This work is licensed under a Creative Commons Attribution-Noncommercial-No Derivative Works 4.0 License.
Included in
Biochemistry, Biophysics, and Structural Biology Commons, Cell and Developmental Biology Commons, Genetics and Genomics Commons, Therapeutics Commons