Recombinant virus products and methods for inhibition of expression of DUX4
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United States of America Patent
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Oct 18, 2016
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Oct 30, 2014
app pub date -
Jul 24, 2012
filing date -
Jul 25, 2011
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Abstract
The present invention relates to RNA interference-based methods for inhibiting the expression of the DUX4 gene, a double homeobox gene on human chromosome 4q35. Recombinant adeno-associated viruses of the invention deliver DNAs encoding microRNAs that knock down the expression of DUX4. The methods have application in the treatment of muscular dystrophies such as facioscapulohumeral muscular dystrophy.
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