Aplicabilidade da terapia gênica no tratamento de pacientes com fibrose cística

Abstract

INTRODUCTION: Cystic fibrosis (CF) is a genetic disease caused by mutation of the gene called cystic fibrosis transmembrane conductance regulator (CFTR). In cystic fibrosis there are structural and functional abnormalities of the CFTR protein, which affects the transport of ions, thus causing dehydration and the production of secretions, also known as mucus. In most cases, it is identified in childhood, through neonatal screening programs, also known as the heel prick test. Through studies carried out over time, research has emerged on the use of gene therapy in the treatment of cystic fibrosis, it is a therapy that consists of changing the basis of the disease and contributes to improving the quality of life of patients. OBJECTIVE: The aim of this study was to analyze the applicability of using gene therapy in patients with cystic fibrosis. METHODOLOGY: Refers to an integrative review, through a bibliographic survey, carried out in PubMed, Scielo, MEDLINE and LILACS databases. FINAL CONSIDERATIONS: In addition to the methods mentioned in this study, it was possible to perceive that there are still limitations in research that need to be discussed, so that this treatment is more used and tested in the future.