Recent progress in gene therapy to deliver nucleic acids with multivalent cationic vectors.
Due to the potential use as transfecting agents of nucleic acids (DNA or RNA), multivalent cationic non-viral vectors have received special attention in the last decade. Much effort has been addressed to synthesize more efficient and biocompatible gene vectors able to transport nucleic acids into the cells without provoking an immune response. Among them, the mostly explored to compact and transfect nucleic acids are: (a) gemini and multivalent cationic lipids, mixed with a helper lipid, by forming lipoplexes; and (b) cationic polymers, polycations, and polyrotaxanes, by forming polyplexes. This review is focused on the progress and recent advances experimented in this area, mainly during the present decade, devoting special attention to the lipoplexes and polyplexes, as follows: (a) to its biophysical characterization (mainly electrostatics, structure, size and morphology) using a wide variety of experimental methods; and (b) to its biological activity (transfection efficacy and cytotoxicity) addressed to confirm the optimum formulations and viability of these complexes as very promising gene vectors of nucleic acids in nanomedicine.
Grupo de Química Coloidal y Supramolecular, Departamento de Química Física I, Facultad de Ciencias Químicas, Universidad Complutense, 28040 Madrid, Spain.
Elena
E
Junquera
Grupo de Química Coloidal y Supramolecular, Departamento de Química Física I, Facultad de Ciencias Químicas, Universidad Complutense, 28040 Madrid, Spain. Electronic address: aicart@quim.ucm.es.
Emilio
E
Aicart
D000818:Animals; D018014:Gene Transfer Techniques; D030342:Genetic Diseases, Inborn / Q000473:pathology / Q000628:therapy; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000737:chemistry* / Q000493:pharmacokinetics; D006801:Humans; D008055:Lipids / Q000737:chemistry / Q000493:pharmacokinetics; D009369:Neoplasms / Q000235:genetics / Q000473:pathology / Q000628:therapy; D009696:Nucleic Acids / Q000493:pharmacokinetics / Q000627:therapeutic use*; D011073:Polyamines / Q000737:chemistry / Q000493:pharmacokinetics; D000071228:Polyelectrolytes; D000644:Quaternary Ammonium Compounds / Q000737:chemistry / Q000493:pharmacokinetics; D043862:Rotaxanes / Q000737:chemistry / Q000493:pharmacokinetics
D008055:Lipids; D009696:Nucleic Acids; D011073:Polyamines; D000071228:Polyelectrolytes; D000644:Quaternary Ammonium Compounds; D043862:Rotaxanes
Cationic polyrotaxanes; DNA; Gemini or multivalent cationic lipids; Gene therapy; Polycations; siRNA