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Journal / Genetics / Entrega sistêmica de MicroRNA utilizando vírus…
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Genetics

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JoVE Journal Genetics
Entrega sistêmica de MicroRNA utilizando vírus Adeno-associado recombinante sorotipo 9 para tratar doenças neuromusculares em roedores
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Entrega sistêmica de MicroRNA utilizando vírus Adeno-associado recombinante sorotipo 9 para tratar doenças neuromusculares em 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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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.

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Genética edição 138 miRNA terapêutica de RNA rAAV terapia genética doença Neuromuscular silenciamento do Gene
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