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Journal / Genetics / Kemirgen nöromusküler hastalıkları tedavi etmek…
JoVE Journal
Genetics

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JoVE Journal Genetics
Kemirgen nöromusküler hastalıkları tedavi etmek için rekombinant Adeno ilişkili virüs serotip 9 kullanarak mikroRNA sistemik teslim
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Kemirgen nöromusküler hastalıkları tedavi etmek için rekombinant Adeno ilişkili virüs serotip 9 kullanarak mikroRNA sistemik teslim

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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Burada bir rekombinant adeno ilişkili virüs serotip 9 nöromuskuler hastalık bir fare model kullanma mikroRNA teslimini açıklayın. Sürekli miRNA overexpression kas ve motor nöronlar, bir fırsat çalışma miRNA işlevi ve tedavi edici potansiyel içinde vivosağlayan bir tek çevresel yönetim farelerde sonuçlandı.

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Genetik sayı 138 miRNA RNA tedavi rAAV gen tedavisi Gene susturmak nöromuskuler hastalık
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