Detalle Publicación

Polycationic amphiphilic cyclodextrin-based nanoparticles for therapeutic gene delivery

Autores: Méndez Ardoy, A.; Urbiola Pérez, Koldo; Aranda Barandiain, Cristina; Ortiz Mellet, C.; García Fernández, J. M.; Tros de Ilarduya Apaolaza, María de la Concepción
Título de la revista: NANOMEDICINE
ISSN: 1743-5889
Volumen: 6
Número: 10
Páginas: 1697 - 1707
Fecha de publicación: 2011
Aim: 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. Materials & 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. Results & Conclusion: 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.