2015/04/09 by Émilie Plantié, Marta Migocka-Patrzałek, Małgorzata Daczewska +1 · 1 citation
Biochemistry, Genetics and Molecular Biology · Medicine · #Muscle Physiology and Disorders #Genetics, Aging, and Longevity in Model Organisms #Cardiomyopathy and Myosin Studies
paper · pdf · doi:10.3390/molecules20046237
openalex publication_date 2015/04/09 · openalex created_date 2025/10/10 · openalex updated_date 2026/07/30
Muscular dystrophies (MD) are a heterogeneous group of genetic disorders that cause muscle weakness, abnormal contractions and muscle wasting, often leading to premature death. More than 30 types of MD have been described so far; those most thoroughly studied are Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and congenital MDs. Structurally, physiologically and biochemically, MDs affect different types of muscles and cause individual symptoms such that genetic and molecular pathways underlying their pathogenesis thus remain poorly understood. To improve our knowledge of how MD-caused muscle defects arise and to find efficacious therapeutic treatments, different animal models have been generated and applied. Among these, simple non-mammalian Drosophila and zebrafish models have proved most useful. This review discusses how zebrafish and Drosophila MD have helped to identify genetic determinants of MDs and design innovative therapeutic strategies with a special focus on DMD, DM1 and congenital MDs.