Plug-and-play assembly of biodegradable ionizable lipids for potent mRNA delivery and gene editing in vivo

Affiliations

• 1 Department of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104, USA.
• 2 Key Laboratory of RNA Innovation, Science and Engineering, CAS Center for Excellence in Molecular Cell Science, Shanghai Institute of Biochemistry and Cell Biology, Chinese Academy of Sciences, University of Chinese Academy of Sciences, Shanghai 200031, China.
• 3 These authors contribute equally to this work.
• 4 Department of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
• 5 Penn Institute for RNA Innovation, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
• 6 School of Pharmacy, East China Normal University, Shanghai 200062, China.
• 7 Division of Pediatric General, Thoracic, and Fetal Surgery, The Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
• 8 Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
• 9 Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
• 10 Institute for Immunology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
• 11 Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
• 12 Institute for Regenerative Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.