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dc.contributor.authorCherry, Jonathan J.
dc.contributor.authorEvans, Matthew C.
dc.contributor.authorNi, Jake
dc.contributor.authorCuny, Gregory D.
dc.contributor.authorGlicksman, Marcie A.
dc.contributor.authorAndrophy, Elliot J.
dc.date.accessioned2020-03-10T17:31:29Z
dc.date.available2020-03-10T17:31:29Z
dc.date.issued2013-12
dc.identifier.citationCopyright 2012 Journal of Biomolecular Screening. This is a post-print version of a published paper that is available at: https://journals.sagepub.com/doi/full/10.1177/1087057111431605. Recommended citation: Cherry, Jonathan J., Matthew C. Evans, Jake Ni, Gregory D. Cuny, Marcie A. Glicksman, and Elliot J. Androphy. "Identification of novel compounds that increase SMN protein levels using an improved SMN2 reporter cell assay." Journal of biomolecular screening 17, no. 4 (2012): 481-495. doi: 10.1177/1087057111431605. This item has been deposited in accordance of publisher copyright and licensing and with the author's permission.
dc.identifier.urihttps://hdl.handle.net/10657/5955
dc.description.abstractSpinal muscular atrophy (SMA) is a neurodegenerative disorder that is characterized by progressive loss of motor neuron function. It is caused by the homozygous loss of the SMN1 (survival of motor neuron 1) gene and a decrease in full-length SMN protein. SMN2 is a nearly identical homolog of SMN1 that, due to alternative splicing, expresses predominantly truncated SMN protein. SMN2 represents an enticing therapeutic target. Increasing expression of full-length SMN from the SMN2 gene might represent a treatment for SMA. We describe a newly designed cell-based reporter assay that faithfully and reproducibly measures full-length SMN expression from the SMN2 gene. This reporter can detect increases of SMN protein by an array of compounds previously shown to regulate SMN2 expression and by the overexpression of proteins that modulate SMN2 splicing. It also can be used to evaluate changes at both the transcriptional and splicing level. This assay can be a valuable tool for the identification of novel compounds that increase SMN2 protein levels and the optimization of compounds already known to modulate SMN2 expression. We present here preliminary data from a high-throughput screen using this assay to identify novel compounds that increase expression of SMN2.
dc.language.isoen_US
dc.publisherJournal of Biomolecular Screening
dc.subjectspinal muscular atrophy
dc.subjectsurvival of motor neuron
dc.subjectSMN1
dc.subjectSMN2
dc.subjectcell-based assay
dc.subjecthigh-content screening
dc.subjectHTS
dc.titleIdentification of Novel Compounds That Increase SMN Protein Levels Using an Improved SMN2 Reporter Cell Assay
dc.typeArticle


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