RNA interference provides researchers with a simple and effective tool to inhibit the function of any human gene. The inherent sequence specificity and lasting activity of small interfering RNAs (siRNAs) makes them ideal drugs, which are transforming drug development and our approach to human health. The promise of RNA interference as a general therapeutic strategy, however, depends on the ability to deliver siRNAs to a wide range of tissues. We believe that modulating the chemistry of RNA is the way to enable delivery to wide range of tissues.
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The focus of the Khvorova Lab is enabling therapeutic oligonucleotide delivery to tissues other than liver through chemical engineering. We are interested in identifying chemical and biological properties that drive siRNA tissue distribution, retention, cellular uptake, and biological availability. By screening a wide range of chemically engineered and naturally occurring bioactive conjugates, we already have identified novel chemical modalities that support delivery of robust amounts of RNAs to the placenta, heart, muscle, kidneys, vasculature, and brain, tissues previously untargetable by RNAi.
