In recent years, gene therapy has emerged as one of the most promising therapeutic avenues for muscular dystrophies. This review synthesizes the latest advances across several modalities, offering a forward-looking assessment of the field.

Key areas covered include:

     Gene replacement strategies, especially for monogenic diseases

     Genome editing technologies, including CRISPR/Cas9, ZFNs, and TALENs

     Gene inactivation approaches to modulate disease pathways

     Delivery systems, with AAV vectors leading clinical translation efforts

     Challenges and ethical considerations in pediatric rare-disease treatment

Importantly, the review highlights how vector selection, tissue tropism, and payload constraints continue to shape therapeutic feasibility — all directly relevant to CHKB deficiency, where skeletal muscle, cardiac tissue, and CNS involvement require a highly adaptable delivery platform.

These insights reinforce the importance of optimized vector engineering and translational biology in bringing safe, effective genetic medicines to patients living with neuromuscular disease.

Stay connected as we continue advancing gene therapy strategies designed for the unique needs of ultra-rare neuromuscular diseases.