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Archivos argentinos de pediatría

Print version ISSN 0325-0075


CAVAGNARI, Brian M. Gene therapy: nucleic acids as drugs. Action mechanisms and delivery into the cell. Arch. argent. pediatr. [online]. 2011, vol.109, n.3, pp.232-236. ISSN 0325-0075.

Gene therapy involves the transference of new genetic material to the cell in order to obtain a therapeutic benefit, offering a new option for the treatment of various diseases. In this article, some of these nucleic acid-based drugs, such as plasmids, aptamers, oligonucleotides, ribozymes and small interfering ribonucleic acid, are presented. Their mechanism and level of action is commented and several delivery systems, such as liposomes, cationic polymers, direct nucleic acid transfer and viral vectors, are also discussed.

Keywords : Gene therapy; Ribozymes; Aptamers; Oligonucleotides; Viral vectors.

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