Medical Research

University of California, San Diego

Steven Dowdy, Yitzhak Tor
San Diego, CA
$1,000,000
June 2012

The discovery of RNA interference (RNAi), a natural gene silencing mechanism with exquisite selectivity for all 23,000 human mRNAs, opened the door to a global therapeutic approach that can be tailored to keep evolutionary pace with pandemic viral outbreaks or for personalized medicine. However, use of RNAi as a therapeutic is severely hindered by the negatively charged phosphate backbone: it simply cannot enter cells on its own. Current RNAi delivery approaches utilize nanoparticles that are 5,000-fold larger than the actual RNAi cargo, which due to this size, have inherently poor pharmacological properties. Using an interdisciplinary chemistry and molecular approach, we seek funds to build a paradigm shifting RNAi technology that radically shrinks the delivery size to the smallest possible, self delivering monomeric RNAi-inducing molecule via synthesis of neutral, bioreversible phosphotriester RiboNucleic Neutrals. Once inside cells, cytoplasmic enzymes unmask the phosphate groups and convert them into negatively charged, active RNAi molecules. Although the technology has broad applicability, the team will establish efficacy of this delivery system in mouse models of cancer.

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