Article

A deep learning approach to identify new gene targets of a novel therapeutic for human splicing disorders

2020-02-04

Abstract excerpt

Pre-mRNA splicing is a key control point in human gene expression. Disturbances in splicing due to mutation or aberrant splicing regulatory networks lead to dysregulated protein expression and contribute to a substantial fraction of human disease. Several classes of active and selective splicing modulator compounds have been recently identified, thus proving that pre-mRNA splicing is a viable target for therapy. W...

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Identifiers and source

Literature Corpus work: e8002a50-9819-5703-955e-1949a13eb43f
DOI: 10.1101/2020.02.03.932103

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