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Journal / Genetics / 重组腺相关病毒9型 MicroRNA…
JoVE Journal
Genetics

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JoVE Journal Genetics
重组腺相关病毒9型 MicroRNA 系统分娩治疗啮齿动物神经肌肉疾病
Visualization of Microbiota in Tick Guts by Whole-mount In Situ Hybridization
Journal (Genetics)
Visualization of Microbiota in Tick Guts by Whole-mount In Situ Hybridization
Artificial RNA Polymerase II Elongation Complexes for Dissecting Co-transcriptional RNA Processing Events
Journal (Genetics)
Artificial RNA Polymerase II Elongation Complexes for Dissecting Co-transcriptional RNA Processing Events
 
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重组腺相关病毒9型 MicroRNA 系统分娩治疗啮齿动物神经肌肉疾病

Article DOI: 10.3791/55724-v • 06:51 min • August 10th, 2018
August 10th, 2018 •
1, 2, 1, 1, 1,3, 1, 1,4
1Neurogenetics Branch, National Institute of Neurological Disorders and Stroke, National Institutes of Health, 2Section on Nervous System Development and Plasticity, The Eunice Kennedy Shriver National Institute of Child and Human Development, National Institutes of Health, 3Department of Molecular Biosciences, Rice Institute for Biomedical Research, Northwestern University, 4Department of Physiology, Anatomy and Genetics, University of Oxford

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在这里, 我们描述了 microRNA 使用重组腺相关病毒9型在小鼠神经肌肉疾病模型中的交付。小鼠的单个外围管理导致肌肉和运动神经元的持续 miRNA 过度表达, 为研究 miRNA 功能和体内的治疗潜力提供了机会。

Tags

遗传学 问题 138 miRNA RNA 疗法 rAAV 基因治疗 基因沉默 神经肌肉疾病
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