Exploiting endocytosis for transfection of mRNA for cytoplasmatic delivery using cationic gold nanoparticles

Gene therapy holds promise to cure various diseases at the fundamental level. For that, efficient carriers are needed for successful gene delivery. Synthetic 'non-viral' vectors, as cationic polymers, are quickly gaining popularity as efficient vectors for transmitting genes. However, they...

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Detalles Bibliográficos
Autores: Gusta, Muriel F., Edel, Michael J., Salazar, Vivian A., Álvarez-Palomo, Belén, Juan, Manel, Broggini, Massimo, Damia, Giovanna, Bigini, Paolo, Corbelli, Alessandro, Fiordaliso, Fabio, Barbul, Alexander, Korenstein, Rafi, Bastús, Neus G., Puntes, Víctor F.
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2023
País:España
Institución:Consejo Superior de Investigaciones Científicas (CSIC)
Repositorio:DIGITAL.CSIC. Repositorio Institucional del CSIC
OAI Identifier:oai:digital.csic.es:10261/336348
Acceso en línea:http://hdl.handle.net/10261/336348
https://api.elsevier.com/content/abstract/scopus_id/85160049059
Access Level:acceso abierto
Palabra clave:Cationic
Gene therapeutics
Gold nanoparticles
Safety
Transfection
Descripción
Sumario:Gene therapy holds promise to cure various diseases at the fundamental level. For that, efficient carriers are needed for successful gene delivery. Synthetic 'non-viral' vectors, as cationic polymers, are quickly gaining popularity as efficient vectors for transmitting genes. However, they suffer from high toxicity associated with the permeation and poration of the cell membrane. This toxic aspect can be eliminated by nanoconjugation. Still, results suggest that optimising the oligonucleotide complexation, ultimately determined by the size and charge of the nanovector, is not the only barrier to efficient gene delivery.