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Introdução: A Distrofia Muscular de Duchenne (DMD) é uma doença genética progressiva caracterizada pela degeneração muscular devido à ausência da proteína distrofina. Este estudo realiza uma revisão bibliográfica enfocando os avanços na terapia gênica para o tratamento da DMD, abordando quatro áreas principais: a eficácia de diversos vetores virais, as variantes genéticas do gene DMD, os riscos de imunogenicidade e as tecnologias emergentes. Metodologia: Utilizando uma abordagem qualitativa, foram analisados artigos e estudos clínicos sobre técnicas como exon-skipping, CRISPR-Cas9, além de terapias baseadas em vetores virais (como AAV) e o uso de oligonucleotídeos antisense (AONs). Referencial Teórico: A revisão evidencia os resultados de eficácia, os desafios técnicos, como a resposta imunológica aos vetores e as limitações no transporte gênico, bem como as potencialidades e obstáculos, incluindo a variabilidade dos resultados, a necessidade de administrações repetidas e questões relacionadas a custo e acessibilidade. Considerações Finais: Considera-se que, apesar do grande potencial da terapia gênica para a DMD, ainda há desafios a serem superados, tanto técnicos quanto sociais. Este artigo oferece uma visão abrangente do estado atual da pesquisa e reforça a importância do contínuo desenvolvimento e aprimoramento dessas abordagens terapêuticas.
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