Nucleic acid therapy has the potential to treat genetic diseases that affect the lungs. Localized delivery of nucleic acid cargos allows for higher doses than are achievable with systemic delivery and avoids both toxicity and off-target effects. One reason nucleic acid therapeutics have not yet achieved clinical approval for delivery to the lungs is that the forces of inhalation degrade the carriers.
Dr. Ghosh’s team has developed a lipid nanoparticle (LNP) formulation containing ionizable lipids that allow for greater stability after aerosolization. The formulation delivers mRNA to the clinically relevant lung epithelial cell within mice lungs and allows for deposition of mRNA throughout simulated human airways. These properties highlight the promise of the LNP to encapsulate therapeutically relevant cargo for inhaled delivery in future studies.
DOI: https://doi.org/10.1002/btm2.10580