DUX4 reduction and muscle function improvement by subcutaneous delivery of gapmer antisense oligonucleotides
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Facioscapulohumeral muscular dystrophy (FSHD) is caused by aberrant expression of double homeodomain protein 4 (DUX4). The disease has no effective treatment. Previously we demonstrated effective DUX4 knockdown in vitro and in vivo using 2'-O-methoxyethyl (2... ...
