Akyürek Eylem Emek, Erba Martina, Dalla Barba Francesco, Sandonà Dorianna, Sacchetto Roberta
Department of Comparative Biomedicine and Food Science, University of Padova, Viale dell'Università 16, Legnaro, 35020 Padova, Italy.
Department of Biomedical Sciences, University of Padova, 35131 Padova, Italy.
Int J Mol Sci. 2025 Sep 10;26(18):8832. doi: 10.3390/ijms26188832.
The zebrafish () has become one of the most popular and valuable model organisms for studying rare neuromuscular diseases. Its unique characteristics, including the high number of offspring produced with each mating, transparent eggs, rapid development, and genetic similarity to humans, make this small vertebrate ideal for investigating complex and rare disorders affecting the skeletal muscle, such as Duchenne Muscular Dystrophy (DMD), Limb Girdle Muscular Dystrophies (LGMDs), and Brody Myopathy (BM). Various zebrafish models, both natural mutants and genetically engineered strains, have been developed to study these conditions. These models enable the deciphering of pathogenetic mechanisms, the real-time monitoring of disease progression, high-throughput drug screening, and the testing of novel therapeutic approaches. As research progresses, zebrafish models are likely to play an increasingly crucial role in unravelling the complexities of rare neuromuscular diseases and developing targeted therapies, offering hope for affected patients.
斑马鱼已成为研究罕见神经肌肉疾病最受欢迎且最具价值的模式生物之一。它具有独特的特征，包括每次交配产生的后代数量众多、卵透明、发育迅速以及与人类的基因相似性，这使得这种小型脊椎动物成为研究影响骨骼肌的复杂罕见疾病（如杜氏肌营养不良症（DMD）、肢带型肌营养不良症（LGMDs）和布罗迪肌病（BM））的理想选择。已经开发了各种斑马鱼模型，包括自然突变体和基因工程菌株，用于研究这些病症。这些模型有助于解读发病机制、实时监测疾病进展、进行高通量药物筛选以及测试新型治疗方法。随着研究的进展，斑马鱼模型在揭示罕见神经肌肉疾病的复杂性和开发靶向治疗方面可能会发挥越来越关键的作用，为受影响的患者带来希望。
