CRISPR-based methods and products for increasing frataxin levels and uses thereof
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United States of America Patent
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Jun 18, 2019
Grant Date -
Jun 21, 2018
app pub date -
Mar 20, 2015
filing date -
Mar 20, 2014
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Abstract
Methods and products (e.g., gRNAs, recombinant fusion proteins, frataxin targeting systems, compositions and kits) are described for increasing frataxin expression/levels in a cell, as well as uses of such methods and products, for example for the treatment of Friedreich ataxia in a subject suffering therefrom.
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