Prospects of gene therapy for the treatment of inherited neuromuscular disorders

Gene therapy represents a promising treatment method for inherited neuromuscular disorders (NMDs), characterized by high genetic and clinical heterogeneity. This approach aims to correct genetic anomalies that are the primary causes of. The use of adeno-associated viruses and other delivery vectors allows for molecular modifications at the DNA level, opening possibilities for radical treatment of diseases such as spinal muscular atrophy and Duchenne muscular dystrophy. Major challenges in gene therapy include issues of immunogenicity, safety, and ethics, which require further research and international collaboration to optimize and enhance therapeutic strategies.