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1. (WO2018057863) SILENCING OF DUX4 BY RECOMBINANT GENE EDITING COMPLEXES

Pub. No.:    WO/2018/057863    International Application No.:    PCT/US2017/052919
Publication Date: Fri Mar 30 01:59:59 CEST 2018 International Filing Date: Sat Sep 23 01:59:59 CEST 2017
IPC: A61K 48/00
C12N 15/00
C07H 21/04
Applicants: UNIVERSITY OF MASSACHUSETTS
Inventors: JONES, Peter, L.
HIMEDA, Charis, L.
JONES, Takako
Title: SILENCING OF DUX4 BY RECOMBINANT GENE EDITING COMPLEXES
Abstract:
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).