Modular design of non-viral vectors with bioactive components.
Inefficient gene delivery continues to limit gene therapy applications to both basic and applied sciences. Approaches for engineering vectors increasingly include bioactive components that bind cellular receptors, disrupt membranes, or enhance nuclear transport. Recently, a novel cationic lipid was developed by modifying the glucocorticoid dexamethasone. This cationic corticosteroid condenses plasmids for gene delivery, while also modulating inflammation. Modular vectors containing bioactive components that target various cellular processes can overcome the barriers limiting gene transfer.
Departments of Chemical and Biological Engineering and Biomedical Engineering, Northwestern University, 2145 Sheridan Rd / E156, Evanston, IL 60208-3120, USA. l-shea@northwestern.edu
Lonnie D
LD
Shea
Tiffany L
TL
Houchin
D021581:Active Transport, Cell Nucleus / Q000502:physiology; D000818:Animals; D001692:Biological Transport / Q000502:physiology; D003907:Dexamethasone / Q000737:chemistry; D004705:Endocytosis / Q000502:physiology; D005229:Fatty Acids, Monounsaturated / Q000737:chemistry; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000008:administration & dosage* / Q000737:chemistry / Q000235:genetics; D006801:Humans; D007425:Intracellular Membranes / Q000378:metabolism; D019913:Nuclear Localization Signals / Q000008:administration & dosage / Q000235:genetics / Q000502:physiology; D010455:Peptides / Q000008:administration & dosage / Q000235:genetics / Q000502:physiology; D010714:Phosphatidylethanolamines / Q000737:chemistry; D010957:Plasmids / Q000008:administration & dosage / Q000235:genetics; D011094:Polyethyleneimine / Q000737:chemistry; D000644:Quaternary Ammonium Compounds / Q000737:chemistry; D011956:Receptors, Cell Surface / Q000378:metabolism; D014157:Transcription Factors / Q000008:administration & dosage / Q000235:genetics / Q000502:physiology; D014162:Transfection / Q000379:methods*
C410262:1,2-dioleoyl-glycero-3-phosphatidyl ethanolamine; D005229:Fatty Acids, Monounsaturated; D019913:Nuclear Localization Signals; D010455:Peptides; D010714:Phosphatidylethanolamines; D000644:Quaternary Ammonium Compounds; D011956:Receptors, Cell Surface; D014157:Transcription Factors; D003907:Dexamethasone; D011094:Polyethyleneimine; C070046:1,2-dioleoyloxy-3-(trimethylammonium)propane