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Genetics

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JoVE Journal Genetics
Consegna sistemica di MicroRNA usando ricombinanti Virus Adeno-associato sierotipo 9 per trattare malattie neuromuscolari in roditori
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Consegna sistemica di MicroRNA usando ricombinanti Virus Adeno-associato sierotipo 9 per trattare malattie neuromuscolari in roditori

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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Qui descriviamo la consegna del microRNA usando un virus ricombinante adeno-associato sierotipo 9 in un modello murino di una malattia neuromuscolare. Una singola somministrazione periferica nei topi ha provocato la sovraespressione di miRNA sostenuta nel muscolo e motoneuroni, offrendo l'opportunità di studio miRNA funzione e potenziale terapeutico in vivo.

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Genetica problema 138 miRNA terapie basate sul RNA rAAV terapia genica silenziamento genico malattia neuromuscolare
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