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Journal / Genetics / Systemisk levering av MicroRNA ved hjelp av…
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Genetics

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JoVE Journal Genetics
Systemisk levering av MicroRNA ved hjelp av rekombinant Adeno-assosiert Virus Serotype 9 til å behandle Neuromuscular sykdommen i gnagere
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Systemisk levering av MicroRNA ved hjelp av rekombinant Adeno-assosiert Virus Serotype 9 til å behandle Neuromuscular sykdommen i gnagere

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 av microRNA bruker en rekombinant adeno-assosiert virus serotype 9 i en musemodell av en neuromuscular sykdommen. En enkelt ekstern administrasjon i mus resulterte i vedvarende miRNA overuttrykte i muskel og motor neurons, gir en mulighet til å studere miRNA funksjon og terapeutiske potensielle i vivo.

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Genetikk problemet 138 miRNA RNA therapeutics rAAV genterapi Gene stanse Neuromuscular sykdommen
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