This laboratory has developed a neutral lipid based vesicle that uses naturally occurring cholesteryl esters to encapsulate and deliver a wide variety of substances, including fluorescein isothiocyanate (FITC) and other small molecules, vancomycin and other antibiotics, insulin and other peptides, IgG antibodies and other proteins as well as plasmid DNA and other nucleic acids. Previous work has shown cholesteryl ester vesicle-mediated delivery of FITC-labeled peptides into various mouse tissues (including brain) after oral administration.  Cholesteryl ester vesicles could therefore be used to orally deliver compounds for which intravenous administration is the only effective dosing route. Particularly exciting is the potential to orally deliver nucleic acid therapeutics. The present study reports preliminary work on the encapsulation and delivery of plasmid DNA encoding Green Fluorescent Protein (GFP), a molecule widely used as a co-transfection marker and to study protein interaction and localization.  Successful transfection of this plasmid results in a cell that displays green fluorescence when excited with light of the appropriate wavelength.  In the present study GFP was encapsulated and the resulting preparations were characterized for vesicle size as well as lipid and DNA content. Cholesteryl ester vesicle delivery of GFP plasmid into several neural cell lines including SHSY5Y, LUHMES and U373 was demonstrated.  These studies suggest the potential for cholesteryl ester vesicle-mediated delivery of treatments for neurodegenerative diseases.

Cholesteryl ester vesicle mediated delivery of nucleic acids into neural cells <i>in vitro</i>

256th National Meeting (2018)

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Cholesteryl ester vesicle mediated delivery of nucleic acids into neural cells in vitro