BME Seminar Series: Dr. Yizhou Dong, OSU
"Development of nanomaterials for mRNA therapeutics and genome editing"
Messenger RNA (mRNA) therapeutics have shown great promise for purpose of expressing functional proteins. However, the efficient and safe delivery of mRNA remains a key challenge for the clinical application of mRNA based therapeutics. Lipid and lipid-like nanoparticles possess the potential for mRNA delivery. Based on our previous experiences, we have designed and synthesized N1,N3,N5-tris(2-aminoethyl)benzene-1,3,5-tricarboxamide (TT). We applied an orthogonal experimental design to investigate the impacts of formulation components on delivery efficiency. TT3 LLNs, a lead material fully recovered the level of human factor IX (hFIX) to normal physiological values in FIX-knockout mice. In addition, we demonstrated that TT3 LLNs were capable of effectively delivering Cas9 mRNA and guide RNA to the mouse liver for genome editing.
Dr. Dong received his Ph.D. degree in pharmaceutical sciences from the University of North Carolina at Chapel Hill (UNC-CH) in 2009 under the supervision of Professor K.-H. Lee. He was a postdoctoral fellow from 2010-2014 in Children’s Hospital Boston at Harvard Medical School and also in the David H. Koch Institute for Integrative Cancer Research at MIT in the laboratory of Professors Robert Langer and Daniel Anderson. He also holds B.S. in pharmaceutical sciences from Peking University, Health Science Center (2002) and M.S. in organic chemistry from Shanghai Institute of Organic Chemistry (2005). He joined the Division of Pharmaceutics and Pharmaceutical Chemistry at The Ohio State University as Assistant Professor in 2014.