Journal Article Preprint Epublish
PubMed: 39041040 DOI: 10.21203/rs.3.rs-4656663/v1
Targeted delivery of TGF-β mRNA to lung parenchyma using one-component ionizable amphiphilic Janus Dendrimers.
Res Sq
Authors: Elena Atochina-Vasserman, Jaclynn Meshanni, Emily Stevenson, Dapeng Zhang, Rachel Sun, Nathan Ona, Erin Reagan, Elena Abramova, Chang-Jiang Guo, Melissa Wilkinson, Ishana Baboo, Yuzi Yang, Liuyan Pan, Devendra Maurya, Virgil Percec, Yongsheng Li, Andrew Gow, Drew Weissman
Affiliations
- Perelman School of Medicine, University of Pennsylvania.
- University of Pennsylvania.
- Rutgers University.
- East China University of Science and Technology.
- University of Pennsylvania Perelman School of Medicine.
- Roy & Diana Vagelos Laboratories, Department of Chemistry, University of Pennsylvania.
Abstract
Current clinical strategies for the delivery of pulmonary therapeutics to the lung are primarily targeted to the upper portions of the airways. However, targeted delivery to the lower regions of the lung is necessary for the treatment of parenchymal lung injury and disease. Here, we have developed an mRNA therapeutic for the lower lung using one-component Ionizable Amphiphilic Janus Dendrimers (IAJDs) as a delivery vehicle. We deliver an anti-inflammatory cytokine mRNA, transforming growth factor-beta (TGF-β), to produce transient protein expression in the lower regions of the lung. This study highlights IAJD's potential for precise, effective, and safe delivery of TGF-β mRNA to the lung. This delivery system offers a promising approach for targeting therapeutics to the specific tissues, a strategy necessary to fill the current clinical gap in treating parenchymal lung injury and disease.
MeSH
Lung, Dendrimers, Lung Injury, Rna, Messenger, Therapeutics