About this Research Topic
For a long time, the treatment of neuromuscular diseases has remained substantially symptomatic. However, thanks to the advent of new technologies, over the last few years there has been a constant growth in therapeutic studies and clinical trials in the field of neuromuscular diseases.
This has led to the possibility of applying innovative therapeutic methods such as gene replacement, gene editing, and antisense oligonucleotide therapy, to specific neuromuscular diseases. In particular, some of the diseases for which these therapies have been developed include spinal muscular atrophy and muscular dystrophies.
This Research Topic aims to collect high-quality manuscripts detailing the most recent advances in the use of gene therapy for the treatment of inherited neuromuscular diseases. With this Research Topic we aim to obtain a comprehensive update on current applications and future prospects of this new therapeutic strategy.
Keywords: Gene Replacement Therapy, Gene Editing, AAV, Antisense Oligonucleotide Therapy, Exon Skipping
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