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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
Chapitres
- 00:04Titre
- 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
Her beskriver vi levering af mikroRNA ved hjælp af en rekombinant adeno-associeret virus serotype 9 i en musemodel af neuromuskulære sygdomme. En enkelt perifere administration i mus resulteret i vedvarende miRNA overekspression i muskel og motoriske neuroner, giver mulighed for undersøgelse miRNA funktion og terapeutiske potentielle in vivo.
