TERAPIA GÊNICA NA DISTROFIA MUSCULAR DE DUCHENNE: AVANÇOS, DESAFIOS E PERSPECTIVAS

Introduction: Duchenne Muscular Dystrophy (DMD) is a progressive genetic disease characterized by muscle degeneration due to the absence of the dystrophin protein. This study conducts a literature review focusing on advances in gene therapy for the treatment of DMD, addressing four main areas: the efficacy of various viral vectors, genetic variants of the DMD gene, immunogenicity risks, and emerging technologies. Methodology: Using a qualitative approach, articles and clinical studies on techniques such as exon-skipping, CRISPR-Cas9, as well as viral vector-based therapies (such as AAV) and the use of antisense oligonucleotides (AONs) were analyzed. Theoretical Framework: The review highlights efficacy results, technical challenges, such as the immune response to vectors and limitations in gene delivery, as well as potential and obstacles, including variability in results, the need for repeated administrations, and issues related to cost and accessibility. Final Considerations: Despite the great potential of gene therapy for DMD, there are still challenges to be overcome, both technical and social. This article provides a comprehensive overview of the current state of research and reinforces the importance of the continued development and improvement of these therapeutic approaches.