Problem
Nucleic acid therapy has the potential to treat genetic diseases that affect the lungs. In order to effectively deliver genetic material to lung cells, gene delivery systems must be able to overcome the mucus barrier and directly target cells harboring the genetic mutation causing the disease state. Past efforts have focused to penetrate the mucus barrier with the use of hydrophilic polymers, which may trigger an immune response and do not necessarily target cell types.
Solution
Dr. Ghosh’s team has identified novel peptide sequences that are able to overcome the mucus barrier and allow uptake into human bronchial epithelial cells. These peptides allow penetration of the mucus barrier, and when attached to a lipid nanoparticle (LNP) mRNA delivery system, enable higher mRNA expression in mucus producing cystic fibrosis primary cells than LNPs without the peptide.
About the inventor
Dr. Ghosh is an Associate Professor of Molecular Pharmaceutics and Drug Delivery at The University of Texas at Austin, with primary research interests in biologically inspired, rational design of biomolecules (e.g., peptides, proteins) and the creation of nanoscale materials for the development of therapeutics in cancer and mucosal-associated diseases. He has published over twenty research papers and has been awarded numerous U.S. patents. The focus of his work has broad applicability in the integration of pharmaceutical sciences with biotechnology, biological engineering, and materials science.
Reference
https://doi.org/10.1101/2023.09.13.557559
