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Journal / Genetics / Systemisk levering af mikroRNA ved hjælp af…
JoVE Journal
Genetics

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JoVE Journal Genetics
Systemisk levering af mikroRNA ved hjælp af rekombinante Adeno-associeret Virus Serotype 9 til behandling af neuromuskulære sygdomme i gnavere
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Systemisk levering af mikroRNA ved hjælp af rekombinante Adeno-associeret Virus Serotype 9 til behandling af neuromuskulære sygdomme i gnavere

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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Her beskriver vi levering af mikroRNA ved hjælp af en rekombinant adeno-associeret virus serotype 9 i en musemodel af neuromuskulære sygdomme. En enkelt perifere administration i mus resulteret i vedvarende miRNA overekspression i muskel og motoriske neuroner, giver mulighed for undersøgelse miRNA funktion og terapeutiske potentielle in vivo.

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Genetik spørgsmålet 138 miRNA RNA therapeutics rAAV genterapi genhæmning neuromuskulære sygdomme
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