Treatment of acute lung injury using one-component LNP to deliver TGFβ mRNA to the lower lung

Introduction: Acute lung injury (ALI) has nearly 200,000 new diagnosis each year and is associated with high morbidity and mortality (1). Treatments for ALI are limited and are not lung localized to the area of injury (2). Specific delivery of therapeutics to the respiratory epithelium is challenging. This study focused on the development of an immunotherapeutic to treat ALI. We utilized one-component Ionizable Amphiphilic Janus Dendrimers (IAJDs) to deliver mRNA. Here, we delivered an anti-inflammatory cytokine TGF-β to produce transient protein expression in the lower respiratory regions of the lung (3).

Wook-Jin Park – Postdoctoral Fellow, Institute for RNA Innovation, University of Pennsylvania Perelman School of Medicine, USA; Rachel Sun – Graduate Student, Environmental and Occupational Health Sciences Institute, Rutgers University, USA; Nathan Ona – Research Associate, Institute for RNA Innovation, University of Pennsylvania Perelman School of Medicine, USA; Dapeng Zhang – Faculty, East China University of Science and Technology, China; Elena Abramova – Research Associate, Environmental and Occupational Health Sciences Institute, Rutgers University, USA; Changjiang Guo – Research Associate, Environmental and Occupational Health Sciences Institute, Rutgers University, USA; Sydni Berkihiser – Research Associate, Institute for RNA Innovation, University of Pennsylvania Perelman School of Medicine, USA; Emily Stevenson – Postdoctoral Fellow, Environmental and Occupational Health Sciences Institute, Rutgers University, USA; Jessica Vasserman – Undergraduate, Institute for RNA Innovation, University of Pennsylvania Perelman School of Medicine, USA; Ishana Baboo – Undergraduate, Institute for RNA Innovation, University of Pennsylvania Perelman School of Medicine, USA; Andrew Gow – Faculty, Environmental and Occupational Health Sciences Institute, Rutgers University, USA; Drew Weissman – Faculty, Institute for RNA Innovation, University of Pennsylvania Perelman School of Medicine, USA; Elena Atochina-Vasserman – Faculty, Institute for RNA Innovation, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA