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AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD.

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Title: AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD.
Authors: Giesige CR; Biomedical Sciences Graduate Program, The Ohio State University, Columbus, Ohio, USA.; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Wallace LM; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Heller KN; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Eidahl JO; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Saad NY; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Fowler AM; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Pyne NK; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Al-Kharsan M; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Rashnonejad A; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Chermahini GA; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Domire JS; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Mukweyi D; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Garwick-Coppens SE; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Guckes SM; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., McLaughlin KJ; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA., Meyer K; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA., Rodino-Klapac LR; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA., Harper SQ; Biomedical Sciences Graduate Program, The Ohio State University, Columbus, Ohio, USA.; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.
Source: JCI insight [JCI Insight] 2018 Nov 15; Vol. 3 (22). Date of Electronic Publication: 2018 Nov 15.
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
Journal Info: Publisher: American Society for Clinical Investigation Country of Publication: United States NLM ID: 101676073 Publication Model: Electronic Cited Medium: Internet ISSN: 2379-3708 (Electronic) Linking ISSN: 23793708 NLM ISO Abbreviation: JCI Insight Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD.
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  Data: <searchLink fieldCode="AU" term="%22Giesige+CR%22">Giesige CR</searchLink>; Biomedical Sciences Graduate Program, The Ohio State University, Columbus, Ohio, USA.; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Wallace+LM%22">Wallace LM</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Heller+KN%22">Heller KN</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Eidahl+JO%22">Eidahl JO</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Saad+NY%22">Saad NY</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Fowler+AM%22">Fowler AM</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Pyne+NK%22">Pyne NK</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Al-Kharsan+M%22">Al-Kharsan M</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Rashnonejad+A%22">Rashnonejad A</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Chermahini+GA%22">Chermahini GA</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Domire+JS%22">Domire JS</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Mukweyi+D%22">Mukweyi D</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Garwick-Coppens+SE%22">Garwick-Coppens SE</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Guckes+SM%22">Guckes SM</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22McLaughlin+KJ%22">McLaughlin KJ</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Meyer+K%22">Meyer K</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Rodino-Klapac+LR%22">Rodino-Klapac LR</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.<br /><searchLink fieldCode="AU" term="%22Harper+SQ%22">Harper SQ</searchLink>; Biomedical Sciences Graduate Program, The Ohio State University, Columbus, Ohio, USA.; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.; Department of Pediatrics, The Ohio State University, Columbus, Ohio, USA.
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  Data: <searchLink fieldCode="JN" term="%22101676073%22">JCI insight</searchLink> [JCI Insight] 2018 Nov 15; Vol. 3 (22). <i>Date of Electronic Publication: </i>2018 Nov 15.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22American+Society+for+Clinical+Investigation%22">American Society for Clinical Investigation </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>101676073 <i>Publication Model: </i>Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>2379-3708 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2223793708%22">23793708 </searchLink><i>NLM ISO Abbreviation: </i>JCI Insight <i>Subsets: </i>MEDLINE
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        Value: 10.1172/jci.insight.123538
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        Text: English
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      – TitleFull: AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD.
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              Text: 2018 Nov 15
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