Article

Combining multi-omics and drug perturbation profiles to identify novel treatments that improve disease phenotypes in spinal muscular atrophy

2019-12-18

Abstract excerpt

<h4>ABSTRACT</h4> Spinal muscular atrophy (SMA) is a neuromuscular disorder caused by loss of survival motor neuron (SMN) protein. While SMN restoration therapies are beneficial, they are not a cure. We aimed to identify novel treatments to alleviate muscle pathology combining transcriptomics, proteomics and perturbational datasets. This revealed potential drug candidates for repurposing in SMA. One of the lead c...

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Literature Corpus work: b174ff62-ebbf-5ca1-8918-63499f9123f1
DOI: 10.1101/2019.12.17.879353

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