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Systemic delivery of a DUX4-targeting antisense oligonucleotide to treat facioscapulohumeral muscular dystrophy
This paper investigates a potential treatment for facioscapulohumeral muscular dystrophy (FSHD), a genetic disorder caused by the improper expression of the DUX4 gene in muscles, leading to muscle weakness and deterioration. Currently, there is no molecular therapy to halt or slow the progression of FSHD. The researchers tested an antisense oligonucleotide (ASO) that targets and reduces the DUX4 transcript in a mouse model (ACTA1-MCM;FLExDUX4) that mimics FSHD. The ASO treatment, …
