因此,深入解析DMD发病早期的分子和细胞内在变化,对于探索疾病进程的调控机制和开发早期治疗方案具有重要意义。 陈永昌/胡苹/季维智团队在Cell发表题为Profound cellular defects attribute to muscular pathogenesis in rhesus monkey model of Duchenne muscular dystrophy的研究论文。研究首次发现在DMD的早期阶段,肌肉退化往往首...
However, to date the efficiency of this strategy is suboptimal. Here we present methods for assessing exon skipping using AON alone or in combination with skip booster in the context of human DMD patient fibroblast derived myotubes and in the mdx mouse model of DMD....
因此,深入解析DMD发病早期的分子和细胞内在变化,对于探索疾病进程的调控机制和开发早期治疗方案具有重要意义。 2024年9月20日,陈永昌/胡苹/季维智团队在Cell发表题为Profound cellular defects attribute to muscular pathogenesis in rhesus mon...
陈永昌/胡苹/季维智团队在Cell发表题为Profound cellular defects attribute to muscular pathogenesis in rhesus monkey model of Duchenne muscular dystrophy的研究论文。研究首次发现在DMD的早期阶段,肌肉退化往往首先表现在肌肉组织的微环境和细胞组成上,而这些变化主要涉及单核细胞群,如免疫细胞(Immune cells)、成纤维/...
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Several strategies, ranging from cell based therapy to small RNA mediated exon skipping have been proposed as an effective therapy for this disease. Experiments in mice model have shown that upregulation of utrophin, the autosomal homologue of dystrophin can compensate dystrophin deficiency and ...
Increased proteasome activity has been implicated in the atrophy and deterioration associated with dystrophic muscles of Duchenne muscular dystrophy (DMD)... Chiao-nan,Joyce,Chen,... - 《Journal of Muscle Research & Cell Motility》 被引量: 12发表: 2014年 加载更多来源...
陈永昌/胡苹/季维智团队在Cell发表题为Profound cellular defects attribute to muscular pathogenesis in rhesus monkey model of Duchenne muscular dystrophy的研究论文。研究首次发现在DMD的早期阶段,肌肉退化往往首先表现在肌肉组织的微环境和细胞组成上,而这些变化主要涉及单核细胞群,如免疫细胞(Immune cells)、成纤维/...
20. Maggio I, Stefanucci L, Janssen JM, Liu J, Chen X, Mouly V, Goncalves MA. Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations. Nucleic Acids Res. 2016; 44:1449–1470 ...
作者: None 摘要: Studies in cell culture and in mice suggest that peptide-modified morpholino oligomers may be useful for treating DMD. Further details on the research, next steps and licensing status are discussed in the article. DOI: 10.1038/scibx.2008.829 年份: 2008 收藏...