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Systemic delivery of a DUX4-targeting antisense oligonucleotide to treat facioscapulohumeral muscular dystrophy
This study evaluates DUX4-targeting antisense oligonucleotide (ASO) therapy for facioscapulohumeral muscular dystrophy (FSHD). Systemic ASO delivery in an FSHD mouse model reduced DUX4 expression, alleviated inflammation and fibrosis, and prevented activation of DUX4 target genes. However, it did not fully restore muscle strength or prevent muscle mass loss. While promising, further optimization of ASO delivery and efficacy is needed for better therapeutic outcomes, especially if applied early in disease progression.
