Prof. Steven Dowdy - Delivery of RNA Therapeutics: The Great Endosomal Escape!
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All macromolecular therapeutics, including ASOs, siRNAs, peptides, proteins, CRISPR, mRNA and non-viral DNA vectors, are taken up into cells by endocytosis. However, <1% to none of the endocytosed therapeutic cargo escapes from the endosome into the cytoplasm and nucleus of the cell. Thus, for all RNA therapeutics, endosomal escape remains the rate-limiting delivery step that prevents their effective use to treat cancer, pandemic influenza, and heart disease. Our research is focused on addressing this problem by developing new chemistry to synthesize novel universal endosomal escape domains (uEEDs) with the goal of enhancing endosomal escape of by 10-fold in the absence of toxicity.
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