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Aberrant splicing in transgenes containing introns, exons, and V5 epitopes: lessons from developing an FSHD mouse model expressing a D4Z4 repeat with flanking genomic sequences.
PLoS One. 2015 Mar 5;10(3):e0118813. doi: 10.1371/journal.pone.0118813. eCollection 2015.
PLoS One. 2015.
PMID: 25742305
Free PMC article.
RNA interference inhibits DUX4-induced muscle toxicity in vivo: implications for a targeted FSHD therapy.
Wallace LM, Liu J, Domire JS, Garwick-Coppens SE, Guckes SM, Mendell JR, Flanigan KM, Harper SQ.
Wallace LM, et al. Among authors: guckes sm.
Mol Ther. 2012 Jul;20(7):1417-23. doi: 10.1038/mt.2012.68. Epub 2012 Apr 17.
Mol Ther. 2012.
PMID: 22508491
Free PMC article.
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Mouse Dux is myotoxic and shares partial functional homology with its human paralog DUX4.
Eidahl JO, Giesige CR, Domire JS, Wallace LM, Fowler AM, Guckes SM, Garwick-Coppens SE, Labhart P, Harper SQ.
Eidahl JO, et al. Among authors: guckes sm.
Hum Mol Genet. 2016 Oct 15;25(20):4577-4589. doi: 10.1093/hmg/ddw287.
Hum Mol Genet. 2016.
PMID: 28173143
Free PMC article.
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AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD.
Giesige CR, Wallace LM, Heller KN, Eidahl JO, Saad NY, Fowler AM, Pyne NK, Al-Kharsan M, Rashnonejad A, Chermahini GA, Domire JS, Mukweyi D, Garwick-Coppens SE, Guckes SM, McLaughlin KJ, Meyer K, Rodino-Klapac LR, Harper SQ.
Giesige CR, et al. Among authors: guckes sm.
JCI Insight. 2018 Nov 15;3(22):e123538. doi: 10.1172/jci.insight.123538.
JCI Insight. 2018.
PMID: 30429376
Free PMC article.
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