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Glutathione hybrid poly (beta-amino ester)-plasmid nanoparticles for enhancing gene delivery and biosafety - ScienceDirect

Trivedi, Nina Horn, Joseph F

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The free base form of (3R,4R,5S)-5-(difluoromethyl)piperidine-3,4-diol has the following structure: and (3R,4R,5S)-5-(difluoromethyl)piperidine-3,4-diol hydrochloride has the structure according to: [0683] In some embodiments, the pharmacological chaperone is a compound described in U.S