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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
Aqui descrevemos a entrega de microRNA utilizando um vírus adeno-associado recombinante sorotipo 9 em um modelo do rato de uma doença neuromuscular. Uma única administração periférica em camundongos resultou em superexpressão de miRNA sustentada no músculo e neurônios motores, oferecendo uma oportunidade de estudo miRNA função e potencial terapêutico em vivo.
