THE USE OF GENE THERAPY IN THE TREATMENT OF CYSTIC FIBROSIS: A SYSTEMATIC REVIEW

Abstract

Gene therapy, which involves the introduction, alteration or correction of genetic material within an individual's cells to treat a disease, has aroused interest within the scientific community, mainly for the treatment of monogenic diseases. The present work aims to address the most relevant information about gene therapy in the treatment of cystic fibrosis (CF). This study is a systematic review and included research published between 2013 and 2023 in the SCIELO, LILACS, MEDLINE and PUBMED databases. 9 articles were highlighted to compose this review. The most relevant information shows that the addition, inactivation and editing of genes are the techniques used in gene therapy, and among the most used nucleases are: zinc-finger nucleases, transcription activator-like effector nucleases and clustered regularly interspaced short palindromic repeats associated with Cas9 nucleases, the latter being the most promising. The advantages and disadvantages depend mainly on the type of vector used to deliver therapeutic genes, with viral vectors standing out for their efficiency in delivering genetic material, however they are more related to immune responses. It is essential to consider ethical issues, such as informed consent, possible adverse effects and equity in access to gene therapy. In short, gene therapy has significant potential in the treatment of CF, paving the way for an effective cure. However, the success of this approach depends not only on scientific advances, but also on carefully addressing ethical issues. Progress in this field is promising, offering hope for a new era in CF treatment.