GENE THERAPY AND THE CHALLENGES OF ITS CLINICAL APPLICATION: PERSPECTIVES IN BIOMEDICINE

• DOI: https://doi.org/10.22533/at.ed.15951325250212

• Palavras-chave: Gene therapy, Viral vectors, CRISPR/Cas9, Biomedical ethics.

• Keywords: Gene therapy, Viral vectors, CRISPR/Cas9, Biomedical ethics.

• Abstract:

  Gene therapy, as an advanced therapeutic technique aimed at correcting genetic mutations associated with hereditary and acquired diseases, represents an important promise in the treatment of various pathologies. Despite the scientific and technological advances seen in recent decades, the clinical application of gene therapy faces significant challenges, both in terms of technical efficacy and ethical and social acceptance. The main objectives of this study were to analyze the scientific and ethical barriers that limit the use of this intervention and to understand the restrictions imposed by the regulatory and clinical context. To achieve these objectives, a literature review was carried out covering scientific publications on gene therapy, with a focus on studies on viral vectors, gene editing methods and analyses of the social and ethical acceptance of the technology. Articles and reviews published between 2000 and 2023 were selected, ranging from advances in technologies such as CRISPR/Cas9 to the most commonly used viral vectors, such as adenoviruses and virusesadeno-associated , with an emphasis on the adverse immune responses that often compromise the effectiveness of treatment. The results indicate that gene therapy has a high therapeutic potential, especially in monogenic diseases, but its full implementation depends on technological innovations that ensure immunological safety and improve the efficiency of the vectors. In addition, germ cell manipulation raises substantial ethical questions, especially in relation to hereditary impact and the associated long-term risks, which emphasizes the need for strict regulation. In conclusion , although gene therapy represents an innovative alternative in the field of medical interventions, overcoming the technical and ethical challenges is crucial for its definitive integration into clinical practice.