Article
Optimized Cholesterol-siRNA Chemistry Improves Productive Loading onto Extracellular Vesicles.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Aug 2018
Haraszti Reka Agnes, Miller Rachael, Didiot Marie-Cecile, Biscans Annabelle, Alterman Julia F, Hassler Matthew R, Roux Loic, Echeverria Dimas, Sapp Ellen, DiFiglia Marian, Aronin Neil, Khvorova Anastasia
Abstract excerpt
Extracellular vesicles are promising delivery vesicles for therapeutic RNAs. Small interfering RNA (siRNA) conjugation to cholesterol enables efficient and reproducible loading of extracellular vesicles with the therapeutic cargo. siRNAs are typically chemically modified to fit an application. However, siRNA chemical modification pattern has not been specifically optimized for extracellular vesicle-mediated...
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