The progress of antisense-based therapies using first generation Morpholino oligonucleotides for Duchenne muscular dystrophy (DMD) is expected to partially restore dystrophin expression and may prolong the lifespan of DMD patients. In a recent issue of The Journal of Pathology, a sophisticated study by Vila et al used a dystrophic mouse model of DMD to demonstrate that Morpholino-induced exon skipping induced dystrophin expression in skeletal muscle and stimulated cell mediated and humoral responses to dystrophin. The study highlights the need to further investigate the autoimmune response against de novo synthesised truncated dystrophin protein and its long-term consequences after exon-skipping therapy for DMD. © 2019 Pathological Society of Great Britain and Ireland. Published by John Wiley & Sons, Ltd.

中文翻译：

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杜兴氏肌营养不良小鼠模型中外显子跳过疗法后的自身免疫反应及其长期后果。

使用第一代吗啉代寡核苷酸治疗杜兴氏肌营养不良症（DMD）的反义疗法的进展有望部分恢复肌营养不良蛋白的表达，并可能延长DMD患者的寿命。在最近出版的《病理学杂志》中，Vila等人进行了一项复杂的研究，使用了DMD的营养不良小鼠模型来证明吗啉代诱导的外显子跳过了骨骼肌中肌营养不良蛋白的表达，并刺激了细胞对肌营养不良蛋白的介导和体液反应。该研究强调需要进一步研究针对从头合成的截短的肌营养不良蛋白的自身免疫反应及其对DMD的外显子跳过治疗后的长期后果。©2019英国和爱尔兰病理学会。由John Wiley＆Sons，Ltd.出版