Autoimmune response and its long‐term consequences after exon‐skipping therapy in a Duchenne muscular dystrophy mouse model
Autoimmune response and its long‐term consequences after exon‐skipping therapy in a Duchenne muscular dystrophy mouse model
复制标题
杜氏肌营养不良症小鼠模型外显子跳跃治疗后的自身免疫反应及其长期后果
DOI：
10.1002/path.5327
复制
发表时间：
2019
期刊：
影响因子：
--
通讯作者：
Aoki Yoshitsugu
中科院分区：
文献类型：
--
作者：
Nordin Joel Z;Aoki Yoshitsugu
使用第一代吗啉代寡核苷酸治疗杜氏肌营养不良症（DMD）的反义疗法的进展预计将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。在最近一期的病理学杂志中，Vilaet的一项复杂的研究使用DMD的营养不良小鼠模型来证明吗啉诱导的外显子跳跃诱导骨骼肌中的肌营养不良蛋白表达，并刺激细胞介导和体液对肌营养不良蛋白的反应。该研究强调了进一步研究针对新合成的截短型肌营养不良蛋白的自身免疫反应及其在DMD外显子跳跃治疗后的长期后果的必要性。© 2019大不列颠和爱尔兰病理学会。出版社：John Wiley & Sons，Ltd
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 ofThe Journal of Pathology, a sophisticated study by Vilaet alused 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 againstde novosynthesised 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.