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Article

Inhibition of DUX4 expression with antisense LNA gapmers as a therapy for facioscapulohumeral muscular dystrophy

29 Jun 2020

Abstract excerpt

Significance Facioscapulohumeral dystrophy (FSHD) is an inherited disabling muscular disorder caused by misexpression of DUX4 in skeletal muscles. FSHD has variable onset; its infantile form has a more severe disease course. There is no cure for FSHD. Here, we show the potential of antisense oligonucleotides called locked nucleic acid (LNA) gapmers for treating FSHD. We designed LNA gapmers to knock down DUX4...

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Identifiers

PMID: 32601200
DOI: 10.1073/pnas.1909649117

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