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Development of a Novel Targeted RNAi Delivery Technology inTherapies for Metabolic Diseases

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Technical Report,30 Sep 2016,29 Sep 2017

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University Of Massachusetts Medical School Worcester United States

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The objective of this project is to develop a novel RNA interference-based therapy for fatty liver diseases and steatohepatitis. Our. Our strategy is to develop a means of targeting siRNA molecules to relevant cells of the liver in order to silence genes that play key roles in inflammatory, metabolic or other pathways that cause disease initiation or progression. The key specific goal of our project is to develop a simplified delivery vehicle for such gene silencing in liver. During this project we have found that covalent coupling of a moiety e.g., cholesterol that promotes entry into cells directly onto the siRNA forming self-delivery sd RNAs rather than directly onto glucan shells that target Kupffer cells is a superb approach. During this past year we have tested several types of hydrophobic molecules, in comparison with cholesterol, covalently attached to the modified siRNA to determine their efficiency for gene silencing in mice.

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  • Medicine and Medical Research

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