Article

A mutation-independent approach via transcriptional upregulation of a disease modifier gene rescues muscular dystrophy <i>in vivo</i>

2018-03-23

Abstract excerpt

<h4>Introductory paragraph</h4> Identification of protective and/or pathogenic genetic modifiers provides important insight into the heterogeneity of disease presentations in individuals affected by neuromuscular disorders (NMDs), despite having well-defined pathogenic variants. Targeting modifier genes to improve disease phenotypes could be especially beneficial in cases where the causative genes are large, stru...

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Identifiers and source

Literature Corpus work: 64477083-bf91-5140-9254-9ffb46a60fed
DOI: 10.1101/286500

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