P.56 Urine titin as a novel biomarker for Duchenne muscular dystrophy
Neuromuscular Disorders(2022)
摘要
•Mdx mice lacking dystrophin have elevated urine titin levels.•Treatment with an exon skipping method rescued dystrophin expression and decreased urine titin in mdx mice.•Urine titin is elevated in patients with DMD relative to healthy subjects.•Elevated urine titin is a characteristic of DMD patients and the mdx mouse model.•Urine titin may serve as a pharmacodynamic marker for dystrophin restoration therapy.
更多查看译文
关键词
Duchenne muscular dystrophy,Urine biomarker,Titin,Mdx mice,Exon skipper
AI 理解论文
溯源树
样例
![](https://originalfileserver.aminer.cn/sys/aminer/pubs/mrt_preview.jpeg)
生成溯源树,研究论文发展脉络
Chat Paper
正在生成论文摘要