Korean J Neuromuscul Disord.  2023 Dec;15(2):31-35. 10.46518/kjnmd.2023.15.2.31.

Gene Therapy for Genetic Muscle Diseases

• Lee J

• KMID: 2550213
• DOI: http://doi.org/10.46518/kjnmd.2023.15.2.31

Abstract

Genetic muscle diseases encompass a group of conditions where genetic alterations affect skeletal muscles, leading to muscle weakness and hypotonia. Presently, the focus of treatment is on managing symptoms rather than addressing the root cause of the disease. However, recent advancements in gene therapy offer potential cure for these diseases, with over 180 genes identified as contributing factors. Encouraging results have emerged from preclinical trials conducted using animal models, prompting the initiation of several clinical trials aimed at assessing the safety and efficacy of gene therapy in human patients. In this review, we aim to provide an overview of the fundamental concept of gene therapy and discuss ongoing clinical trials that hold promise for curing genetic muscle diseases.

Keyword

Gene therapy; Muscular disease; Myotonic dystrophy