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Genetics

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JoVE Journal Genetics
Entrega sistémica de microARN mediante Virus Adeno-asociado recombinante serotipo 9 para tratar enfermedades neuromusculares en roedores
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Entrega sistémica de microARN mediante Virus Adeno-asociado recombinante serotipo 9 para tratar enfermedades neuromusculares en roedores

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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Aquí describimos la entrega de microRNA usando un serotipo de virus adeno-asociado recombinante 9 en un modelo de ratón de una enfermedad neuromuscular. Una sola administración periférica en ratones dio lugar a la sobreexpresión de miRNA sostenida en el músculo y neuronas del motor, proporcionando una oportunidad de estudio miRNA función y potencial terapéutico en vivo.

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Genética número 138 miRNA terapéutica de RNA rAAV terapia génica enfermedad Neuromuscular de silenciamiento del gen
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