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SILENCING OF DUX4 BY RECOMBINANT GENE EDITING COMPLEXES
SILENCING OF DUX4 BY RECOMBINANT GENE EDITING COMPLEXES
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机译:重组基因编辑复合物对DUX4的沉默
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摘要
The disclosure relates to methods and compositions for regulating expression of DUX4. Specifically, the disclosure provides a recombinant gene editing complex comprising: a recombinant gene editing protein; and, a nucleic acid encoding a guide RNA (gRNA) that specifically hybridizes to a target nucleic acid sequence encoding a D4Z4 macrosatellite repeat region, wherein binding of the complex to the target nucleic acid sequence results in inhibition of DUX4 gene expression. In some aspects, methods described by the disclosure are useful for treating a disease associated with aberrant DUX4 expression (e.g., facioscapulohumeral muscular dystrophy, FSHD).
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