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1. (WO2013120038) MORPHOLINO TARGETING DUX4 FOR TREATING FSHD

Pub. No.:    WO/2013/120038    International Application No.:    PCT/US2013/025461
Publication Date: Fri Aug 16 01:59:59 CEST 2013 International Filing Date: Sun Feb 10 00:59:59 CET 2013
IPC: C12N 15/11
C07H 21/04
Applicants: REGENTS OF THE UNIVERSITY OF MINNESOTA
KYBA, Michael
MULTHAUP, Megan
Inventors: KYBA, Michael
MULTHAUP, Megan
Title: MORPHOLINO TARGETING DUX4 FOR TREATING FSHD
Abstract:
A composition and method is provided that comprises a morpholino oligonucleotide capable of inhibiting DUX4 expression. The morpholino oligonucleotide can provide a therapeutic effect in individuals with facioscapulohumeral muscular dystrophy. Further, the composition and method may comprise a dendrimer delivery moiety.