Article

Quantifying anti-DUX4 therapy for facioscapulohumeral muscular dystrophy

2024-08-15

Abstract excerpt

Facioscapulohumeral muscular dystrophy (FSHD) is an inherited skeletal myopathy with no cure. Expression of the myotoxic transcription factor double homeobox 4 ( DUX4 ) is believed to underlie FSHD pathogenesis and many proposed therapies target DUX4 generation or function. Which of these therapies will be the most effective is unclear. Here, by constructing a Markov-chain-based mathematical model of DUX4-mediate...

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Literature Corpus work: 6b80fa3b-b544-5260-a49a-16e95a004676
DOI: 10.1101/2024.08.14.607973

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