Bridging science and hope: the evolving story of gene therapy for neuromuscular diseases

The field of gene therapy for neuromuscular dystrophies has evolved over the past two decades. Despite some outstanding positive outcomes, some unfortunate adverse effects also led to big setbacks. One important key point is to study relevant preclinical models and to embrace diverse strategies to mitigate or avoid such negative outcomes. Although at first, for some diseases, the promise of a one-treatment-for-all approach was envisioned, it has recently become clear that a personalized approach will likely be preferable given the high variability in response between individuals.