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Hit-and-run silencing of endogenous DUX4 by targeting DNA hypomethylation on D4Z4 repeats in facioscapulohumeral muscular dystrophy

2022-04-12

Abstract excerpt

Facioscapulohumeral muscular dystrophy (FSHD), a progressive skeletal muscle disorder, is epigenetically characterized by DNA hypomethylation of the D4Z4 repeats in the 4q35 region, which enables aberrant DUX4 expression. Sustainable DUX4 suppression is thus a promising therapeutic strategy by which to prevent disease progression, but most of the supposed methods to achieve this depend on the expression of a med...

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Literature Corpus work: cc9dffab-232e-537f-b596-adc99742f5e0
DOI: 10.1101/2022.04.12.487997

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Hit-and-run silencing of endogenous DUX4 by targeting DNA hypomethylation on D4Z4 repeats in facioscapulohumeral muscular dystrophyDOI 10.1101/2022.04.12.487997Read

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Hit-and-run silencing of endogenous <i>DUX4</i> by targeting DNA hypomethylation on D4Z4 repeats in facioscapulohumeral muscular dystrophy

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