Work

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

Year: 2026

Type: article

Abstract: 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.

Source: Frontiers in Cell and Developmental Biology

DOI: 10.3389/fcell.2026.1765367

Authors: Nicolas Wein, Florian Barthélémy

Institutions: Inserm, Centre Hospitalier Universitaire de Nantes, Translational Research in Gene Therapy, Nantes Université, University of California, Los Angeles

Language: English

Topic: Neurogenetic and Muscular Disorders Research

Subfield: Genetics

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,801