Development of pharmacodynamic biomarkers for Duchenne muscular dystrophy using exon skipping treatment and mdx mouse model.

机译：外显子跳跃治疗和MDX小鼠模型的Duchenne肌营养不良药物动力学生物标志物的发展。

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SILAC mouse strategy in combination with mass spectrometry analysis enabled accurate monitoring of biomarkers levels in PMO treated mdx-23 mice. This led to the identification of potential pharmacodynamic biomarkers indicative of restoration of dystrophin expression in dyhstrohin deficient skeletal muscle. These biomarkers need to be tested for their clinical value in future DMD clinical trials dealing with dystrophin replacement therapies.

机译：氧化硅鼠策略与质谱分析相结合，使PMO处理的MDX-23小鼠中的生物标志物水平的精确监测能够精确监测。这导致鉴定潜在的药效学士生物标志物，其恢复Dyhhin缺乏骨骼肌中的肌营养蛋白表达。这些生物标志物需要在处理营养不良替代疗法的未来DMD临床试验中进行临床价值。

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《ASMS Conference on Mass Spectrometry and Allied Topics》|2015年||共1页
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Haeri Seol; Meng H Han; Aiping Zhang; Kristy J Brown; Margaret E. Benny Klimek; Maria Candida Vila; Kanneboyina Nagaraju; Eric P Hoffman; Yetrib Hathout;
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1. Effect of different dosing regimens of exon skipping antisense oligonucleotides on functional outcome in the mdx mouse model of Duchenne muscular dystrophy [J] . Datson N., Weij R., Bijl S., Neuromuscular disorders: NMD . 2015,第Suppla2期

机译：外显子跳过反义寡核苷酸的不同给药方案对Duchenne肌营养不良症mdx小鼠模型功能结局的影响
2. Development of Antisense-Mediated Exon Skipping as a Treatment for Duchenne Muscular Dystrophy [J] . Hans Heemskerk, Christa L. de Winter, Gert-Jan B. van Ommen, Annals of the New York Academy of Sciences . 2009,第期

机译：反义介导外显子跳跃治疗杜氏肌营养不良症的研究进展
3. Exon-skipped dystrophins for treatment of Duchenne muscular dystrophy: mass spectrometry mapping of most exons and cooperative domain designs based on single molecule mechanics. [J] . Krieger CC, Bhasin N, Tewari M, Cytoskeleton . 2010,第12期

机译：用于治疗Duchenne肌营养不良症的外显子营养不良蛋白：大多数外显子的质谱图绘制和基于单分子力学的合作域设计。
4. Development of pharmacodynamic biomarkers for Duchenne muscular dystrophy using exon skipping treatment and mdx mouse model. [C] . Haeri Seol, Meng H Han, Aiping Zhang, ASMS Conference on Mass Spectrometry and Allied Topics . 2015

机译：外显子跳跃治疗和MDX小鼠模型的Duchenne肌营养不良药物动力学生物标志物的发展。
5. Experimental and Computational Studies of Structural Differences between Alternative Exon Skipped Repairs for Duchenne Muscular Dystrophy [D] . Ma, Manyuan 2018

机译：实验和计算研究杜兴氏肌营养不良的其他外显子跳过修复之间的结构差异。
6. Oxidative damage to urinary proteins from the GRMD dog and mdx mouse as biomarkers of dystropathology in Duchenne muscular dystrophy [O] . Jessica R. Terrill, Basma A. Al-Mshhdani, Marisa N. Duong, 2020

机译：来自GRMD狗的尿性蛋白和MDX小鼠的氧化损伤作为Dychenne肌营养不良患者Dystrom病变的生物标志物
7. Exon-skipped dystrophins for treatment of Duchenne muscular dystrophy: Mass spectrometry mapping of most exons and cooperative domain designs based on single molecule mechanics [O] . Christine Carag Krieger, Nishant Bhasin, Manorama Tewari, 2010

机译：用于治疗Duchenne肌营养不良的外显子营养不良症：基于单分子力学的大多数外显子和合作域设计的质谱映射

Development of pharmacodynamic biomarkers for Duchenne muscular dystrophy using exon skipping treatment and mdx mouse model.

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