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There is now a CONTENT FREEZE for Mercury while we switch to a new platform. It began on Friday, March 10 at 6pm and will end on Wednesday, March 15 at noon. No new content can be created during this time, but all material in the system as of the beginning of the freeze will be migrated to the new platform, including users and groups. Functionally the new site is identical to the old one. webteam@gatech.edu
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“Platform Technologies for Targeted in vivo Gene Editing”
James Dahlman, Ph.D.
Postdoctoral Fellow
Boad Institute of MIT and Harvard
Seminar will be made available via videoconference in the Health Sciences Research Building, room E 160 and Technology Enterprise Park, room 104.
Once viewed as a passive link between DNA and protein, RNAs are now known to actively modify gene expression. For example, siRNAs temporarily can reduce the expression of any protein, miRNAs can suppress the translation of many genes simultaneously, and CRISPR-Cas9 enables scientists to make targeted and permanent changes in genomic DNA.
Because RNAs can be used to manipulate many genes simultaneously, they could revolutionize the way we study and treat disease. However, the full scientific and clinical potential of RNA is currently limited because we cannot deliver RNA to the right cells in vivo. In vivo delivery is challenging; RNA must be (a) protected from degradation, (b) delivered to the correct tissue, and (c) ferried into the right cell, without setting off an immune response. While nanoparticles have delivered RNA to the liver, delivery to other cells has remained difficult.
In this presentation, I will describe new tools for in vivo RNA delivery and gene editing, developed by integrating chemical engineering, nanotechnology, and biology. One tool, a nanoparticle named 7C1, has delivered RNA to the heart and lung at very low doses, has delivered five different RNAs concurrently in vivo, is being considered for clinical trials, and has been used by ten labs across the United States to study genes associated with cancer, heart disease, and lung disease. Unlike other nanoparticles, 7C1 does not transfect hepatocytes in vivo. We believe that 7C1, which has been described in PNAS, Nature Nanotechnology, and Cell, can be used to make targeted changes in gene expression. I will also describe a high throughput in vivo screening platform for targeted drug delivery. Typically, thousands of drug delivery materials are synthesized and screened for activity in vitro. Only a small fraction is tested in vivo, despite the fact that the in vitro environment does not simulate the complex hurdles that impede delivery in a living animal. To circumvent this, I designed a platform to quantify how well thousands of drug delivery candidates work in vivo. This platform is perfectly suited to (a) identify new materials for cell-specific delivery, and (b) study the relationship between chemical structure and biological function.
Faculty Host: Wilbur Lam, Ph.D.
