ADVANCEMENTS IN GENE THERAPY FOR GENETIC DISORDERS

• DOI: https://doi.org/10.22533/at.ed.2572410072

• Palavras-chave: Terapia gênica. Desordens genéticas. Fibrose cística. Distrofia muscular.

• Keywords: Gene therapy. Genetic disorders. Cystic fibrosis. Muscular dystrophy.

• Abstract: Genetic disorders, caused by mutations in the human genome, present major challenges to healthcare systems worldwide, significantly impacting individuals' quality and life expectancy. This article reviews the efficacy of gene therapy, an innovative approach aimed at correcting or replacing faulty genes, particularly in diseases such as cystic fibrosis and muscular dystrophy. The methodology included analyzing recent clinical studies, which demonstrated significant successes, such as improvements in patients' visual and motor functions. The results highlight the potential of gene therapy to transform the prognosis of these diseases, offering long-term or even permanent solutions. However, despite advancements, challenges like accessibility, equity, and genetic privacy need to be addressed to ensure that all patients can benefit from this transformative technology. It is concluded that gene therapy has immense potential to revolutionize the treatment of genetic disorders, but continuous efforts are needed to overcome ethical and practical barriers.