RNA Interference Inhibits DUX4-induced Muscle Toxicity In Vivo: Implications for a Targeted FSHD Therapy
Wallace, Lindsay M, Liu, Jian, Domire, Jacqueline S, Garwick-Coppens, Sara E, Guckes, Susan M, Mendell, Jerry R, Flanigan, Kevin M, Harper, Scott Q.
Published in Molecular therapy (01.07.2012)
Published in Molecular therapy (01.07.2012)
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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
Ansseau, Eugénie, Domire, Jacqueline S, Wallace, Lindsay M, Eidahl, Jocelyn O, Guckes, Susan M, Giesige, Carlee R, Pyne, Nettie K, Belayew, Alexandra, Harper, Scott Q
Published in PloS one (05.03.2015)
Published in PloS one (05.03.2015)
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Mouse Dux is myotoxic and shares partial functional homology with its human paralog DUX4
Eidahl, Jocelyn O, Giesige, Carlee R, Domire, Jacqueline S, Wallace, Lindsay M, Fowler, Allison M, Guckes, Susan M, Garwick-Coppens, Sara E, Labhart, Paul, Harper, Scott Q
Published in Human molecular genetics (15.10.2016)
Published in Human molecular genetics (15.10.2016)
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AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD
Giesige, Carlee R, Wallace, Lindsay M, Heller, Kristin N, Eidahl, Jocelyn O, Saad, Nizar Y, Fowler, Allison M, Pyne, Nettie K, Al-Kharsan, Mustafa, Rashnonejad, Afrooz, Chermahini, Gholamhossein Amini, Domire, Jacqueline S, Mukweyi, Diana, Garwick-Coppens, Sara E, Guckes, Susan M, McLaughlin, K John, Meyer, Kathrin, Rodino-Klapac, Louise R, Harper, Scott Q
Published in JCI insight (15.11.2018)
Published in JCI insight (15.11.2018)
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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: e0118813
Ansseau, Eugenie, Domire, Jacqueline S, Wallace, Lindsay M, Eidahl, Jocelyn O, Guckes, Susan M, Giesige, Carlee R, Pyne, Nettie K, Belayew, Alexandra, Harper, Scott Q
Published in PloS one (01.03.2015)
Published in PloS one (01.03.2015)
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