ABSTRACT
CRISPR/Cas gene editing and messenger RNA (mRNA)-based protein replacement therapy hold tremendous potential to effectively treat disease-causing mutations with diverse cellular origin. However, it is challenging to rationally design nanoparticles that selectively target specific tissues. In this presentation, I will describe a strategy termed Selective ORgan Targeting (SORT) wherein lipid nanoparticles (LNPs) are systematically engineered to exclusively edit extrahepatic tissues via addition of a supplemental SORT molecule. Lung-, spleen-, and liver-targeted SORT LNPs selectively edited therapeutically relevant cell types including epithelial cells, endothelial cells, B cells, T cells, and hepatocytes. SORT is compatible with multiple gene editing techniques, including mRNA, Cas9 mRNA / sgRNA, and Cas9 ribonucleoprotein (RNP) complexes. It is envisioned that SORT may aid development of protein replacement and gene correction therapeutics in targeted tissues.
 

BIOGRAPHY
Dr. Daniel J. Siegwart is an Associate Professor in the Department of Biochemistry at UT Southwestern Medical Center. He received a B.S. in Biochemistry from Lehigh University (2003), and a Ph.D. in Chemistry from Carnegie Mellon University (2008) with University Professor Krzysztof Matyjaszewski. He also studied as a Research Fellow at the University of Tokyo with Professor Kazunori Kataoka (2006). He then completed a Postdoctoral Fellowship at MIT with Institute Professor Robert Langer and Professor Daniel G. Anderson (2008-2012).
 

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