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Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
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August 10, 2018
DOI:
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
Summary
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Here we describe the delivery of microRNA using a recombinant adeno-associated virus serotype 9 in a mouse model of a neuromuscular disease. A single peripheral administration in mice resulted in sustained miRNA overexpression in muscle and motor neurons, providing an opportunity to study miRNA function and therapeutic potential in vivo.
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Pourshafie, N., Lee, P. R., Chen, K., Harmison, G. G., Bott, L. C., Fischbeck, K. H., Rinaldi, C. Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents. J. Vis. Exp. (138), e55724, doi:10.3791/55724 (2018).
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