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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
Chapters
- 00:04Title
- 00:48Tail Vein Injection of AAV-miRNA Plasmid
- 02:14Behavioral Assays: Hanging Wire and Foot Print Assay
- 03:30Tissue Harvest
- 04:27Results: Treatment of SBMA Mice with miRNA
- 05:59Conclusion
Här beskriver vi leveransen av mikroRNA använda en rekombinant adeno-associerade virus serotyp 9 i en musmodell av en neuromuskulär sjukdom. En perifer administrering hos möss medförde varaktiga miRNA överuttryck i muskel och motoriska nervceller, vilket ger en möjlighet till studie miRNA funktion och terapeutiska potential i vivo.
