Researcher Finds Method to Increase Delivery Efficiency of mRNA Encoding

11/17/2015
Yizhou Dong

Systemic delivery of mRNA-based therapeutics remains a challenging issue for preclinical and clinical studies. Yet, new lipid-like nanoparticles (TT-LLNs) optimized through an orthogonal array design, demonstrates improved delivery efficiency of mRNA encoding luciferase in vitro by over 350-fold with significantly reduced experimental workload. So says Yizhou Dong, PhD, assistant professor at The Ohio State University College of Pharmacy Division of Pharmaceutics and Pharmaceutical Chemistry.

His findings, along with those of his colleagues, have been published in Nano Letters, a monthly peer-reviewed scientific journal published by the American Chemical Society.

“An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo,” explores Messenger RNA (mRNA) based therapeutics and its potential for expressing functional proteins. One optimized TT3 LLN, termed O-TT3 LLNs, was able to restore the human factor IX (hFIX) level to normal physiological values in FIX-knockout mice. Consequently, these mRNA based nanomaterials merit further development for therapeutic applications.

Said Dong: “We are very excited to develop efficient mRNA delivery systems for potential treatment of genetic disorders. We look forward to conducting more profound preclinical studies and moving the project forward.”

Other authors in the article include Bin Li, Xiao Luo and Junfeng Wang, Division of Pharmaceutics and Pharmaceutical Chemistry at Ohio State’s College of Pharmacy; Binbin Deng and David W. McComb, Center for Electron Microscopy and Analysis, Department of Materials Science at Ohio State’s College of Engineering; Yimin Shi and Karin M. L. Gaensler, Department of Medicine, University of California-San Francisco; Xu Tan, Tsinghua-Peking Center for Life Sciences, School of Medicine, Tsinghua University, Beijing; and Amy L. Dunn and Bryce A. Kerlin, Division of Hematology/Oncology/BMT, at Nationwide Children’s Hospital and the Department of Pediatrics, at Ohio State’s College of Medicine.