title: Polycationic amphiphilic cyclodextrin-based nanoparticles for therapeutic gene delivery.
abstract: OBJECTIVE
  In this study, a set of polycationic amphiphilic cyclodextrins featuring self-assembling capabilities in the presence of nucleic acids have been evaluated as therapeutic gene vectors for in vivo purposes.
  
  
  METHODS
  A tetradecacationic structure incorporating 14 primary amino groups and 7 thioureido groups in the primary face of the cyclooligosaccharide core and 14 hexanoyl chains in the secondary face was judged to be optimal for therapeutic gene delivery.
  
  
  CONCLUSIONS
  This compound efficiently mediated serum-resistant transfection in HeLa and HepG2 cells, comparing favorably with branched poly(ethyleneimine), with a low associated toxicity. Further transfection experiments using an encoding therapeutic gene plasmid (pCMVIL-12) were effected to report expression levels of IL-12. Finally, in vivo gene delivery experiments by systemic injection in mice indicated relatively high transfection levels in the liver, overcoming trapping of the nanoparticles in lung cells, with low toxicity.
authors: Department of Organic Chemistry, Faculty of Chemistry, University of Seville, C/Prof. García González 1, E-41002, Seville, Spain.
authors: Alejandro
authors: A
authors: Méndez-Ardoy
authors: Koldo
authors: K
authors: Urbiola
authors: Cristina
authors: C
authors: Aranda
authors: Carmen
authors: C
authors: Ortiz-Mellet
authors: José Manuel
authors: JM
authors: García-Fernández
authors: Conchita Tros
authors: CT
authors: de Ilarduya
mesh_terms: D000818:Animals; D002460:Cell Line; D002470:Cell Survival / Q000187:drug effects; D003505:Cyclodextrins / Q000138:chemical synthesis / Q000737:chemistry* / Q000378:metabolism / Q000633:toxicity; D004247:DNA / Q000235:genetics / Q000378:metabolism*; D018014:Gene Transfer Techniques / Q000295:instrumentation*; D006801:Humans; D051379:Mice; D053758:Nanoparticles / Q000737:chemistry*; D010957:Plasmids / Q000235:genetics / Q000378:metabolism*; D011073:Polyamines / Q000138:chemical synthesis / Q000737:chemistry* / Q000378:metabolism / Q000633:toxicity; D000071228:Polyelectrolytes; D014162:Transfection / Q000379:methods
chemical_list: D003505:Cyclodextrins; D004247:DNA; D011073:Polyamines; D000071228:Polyelectrolytes