Abstract

Gene therapy strategies have become involved in methods for the treatment of inherited or acquired diseases with the aim to correct the faulty genetic code or to modify gene expression. In order to develop safe and effective therapeutic methods for applying in humans, different nucleic acids, termed as vectors, have been engineered, as therapeutic molecules in addition to classical drugs. In this review we included an overview of recent research and patents on therapeutic nucleic acids, designed for use on safe and effective gene therapy protocols for the treatment of diseases, whose results have been obtained in animal models, and which suggests that the use of nucleic acids as therapeutics molecules holds great promise for the future of disease control or cure.

Keywords: AAT deficiency, cancer, childhood cerebral adrenoleukodystrophy, gene therapy, haemophilia, inherited retinal degenerations, non-viral vector, nucleic acids, rheumatoid arthritis, viral vector.