{"645988":{"#nid":"645988","#data":{"type":"event","title":"Selective ORgan Targeting (SORT): A strategy for effective tissue specific mRNA delivery and CRISPR\/Cas gene editing","body":[{"value":"\u003Cp\u003E\u003Cstrong\u003EPRESENTED BY\u003Cbr \/\u003E\r\nDr. Daniel Siegwart\u003C\/strong\u003E\u003Cbr \/\u003E\r\nAssociate Professor, Department of Biochemistry\u003Cbr \/\u003E\r\nUT Southwestern Medical Center\u003Cbr \/\u003E\r\n\u0026nbsp;\u003C\/p\u003E\r\n\r\n\u003Cp\u003E\u003Cstrong\u003EABSTRACT\u003C\/strong\u003E\u003Cbr \/\u003E\r\nCRISPR\/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.\u003Cbr \/\u003E\r\n\u0026nbsp;\u003C\/p\u003E\r\n\r\n\u003Cp\u003E\u003Cstrong\u003EBIOGRAPHY\u003C\/strong\u003E\u003Cbr \/\u003E\r\nDr. 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).\u003Cbr \/\u003E\r\n\u0026nbsp;\u003C\/p\u003E\r\n\r\n\u003Cp\u003E\u003Ca href=\u0022https:\/\/bluejeans.com\/231929118\/3475\u0022 target=\u0022_blank\u0022\u003E\u003Cstrong\u003EClick here to join the seminar via BlueJeans.\u003C\/strong\u003E\u003C\/a\u003E\u003C\/p\u003E\r\n","summary":null,"format":"limited_html"}],"field_subtitle":"","field_summary":[{"value":"\u003Cp\u003EPresented by Dr. Daniel Siegwart, UT Southwestern Medical Center\u003C\/p\u003E\r\n","format":"limited_html"}],"field_summary_sentence":[{"value":"Presented by Dr. Daniel Siegwart, UT Southwestern Medical Center"}],"uid":"27446","created_gmt":"2021-03-31 21:04:26","changed_gmt":"2021-03-31 21:04:26","author":"Joshua Stewart","boilerplate_text":"","field_publication":"","field_article_url":"","field_event_time":{"event_time_start":"2021-04-09T13:00:00-04:00","event_time_end":"2021-04-09T14:00:00-04:00","event_time_end_last":"2021-04-09T14:00:00-04:00","gmt_time_start":"2021-04-09 17:00:00","gmt_time_end":"2021-04-09 18:00:00","gmt_time_end_last":"2021-04-09 18:00:00","rrule":null,"timezone":"America\/New_York"},"extras":[],"related_links":[{"url":"https:\/\/bluejeans.com\/231929118\/3475","title":"Click to join via BlueJeans"}],"groups":[{"id":"1292","name":"Parker H. Petit Institute for Bioengineering and Bioscience (IBB)"},{"id":"1254","name":"Wallace H. Coulter Dept. of Biomedical Engineering"}],"categories":[],"keywords":[],"core_research_areas":[],"news_room_topics":[],"event_categories":[{"id":"1795","name":"Seminar\/Lecture\/Colloquium"}],"invited_audience":[{"id":"78761","name":"Faculty\/Staff"},{"id":"177814","name":"Postdoc"},{"id":"78771","name":"Public"},{"id":"174045","name":"Graduate students"},{"id":"78751","name":"Undergraduate students"}],"affiliations":[],"classification":[],"areas_of_expertise":[],"news_and_recent_appearances":[],"phone":[],"contact":[],"email":[],"slides":[],"orientation":[],"userdata":""}}}