Gene therapy has shown significant potential in the treatment of neuromuscular diseases such as Duchenne muscular dystrophy and spinal muscular atrophy. Advances in viral vector technology and gene editing techniques such as CRISPR-Cas9 have opened up new therapeutic possibilities. This paper explores the main advances, clinical outcomes and challenges associated with the application of this therapeutic approach. The literature review is based on recent studies on innovations in the field of gene therapy.
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Dias et al. (2024) studied this question.
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