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Status |
Public on May 14, 2020 |
Title |
Combining multi-omics and drug perutbation profiles to identify novel treatments that improve disease phenotypes in spinal muscular atrophy [RNA-seq] |
Organism |
Mus musculus |
Experiment type |
Expression profiling by high throughput sequencing
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Summary |
We used an in-depth and parallel approach combining proteomics, transcriptomics and the drug pertubational dataset Connectivity Map (CMap) to identify differentially expressed (DE) transcripts and proteins in skeletal muscle of the severe Taiwanese Smn-/-;SMN2 SMA mice that could potentially be restored by known and available pharmacological compounds. This strategy uncovered several potential therapeutic candidates, including harmine, which was further evaluated in cell and animal models, showing an ability to restore molecular networks and improve several disease phenotypes, including SMN expression and lifespan.
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Overall design |
Examination of harmine treatment effect by comparison of three untreated SMA mice, three treated SMA mice, three untreated control mice and three treated control mice.
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Contributor(s) |
Meijboom KE, Volpato V, Monzón-Sandoval J, Hoolachan JM, Hammond SM, Abendroth F, Gerrit de Jong O, Hazell G, Ahlskog N, Wood MJ, Webber C, Bowerman M |
Citation(s) |
34236053 |
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Submission date |
May 13, 2020 |
Last update date |
Sep 14, 2021 |
Contact name |
Viola Volpato |
E-mail(s) |
volpatov@cardiff.ac.uk
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Organization name |
Cardiff University
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Department |
UK DRI
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Street address |
Maindy Rd
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City |
Cardiff |
ZIP/Postal code |
CF24 4HQ |
Country |
United Kingdom |
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Platforms (1) |
GPL21103 |
Illumina HiSeq 4000 (Mus musculus) |
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Samples (12)
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This SubSeries is part of SuperSeries: |
GSE150563 |
Combining multi-omics and drug perutbation profiles to identify novel treatments that improve disease phenotypes in spinal muscular atrophy |
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Relations |
BioProject |
PRJNA632614 |
SRA |
SRP261500 |