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Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD.

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Title: Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD.
Authors: Wallace LM; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Saad NY; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Pyne NK; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Fowler AM; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Eidahl JO; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Domire JS; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Griffin DA; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Herman AC; Research Information Solutions and Innovation Infrastructure, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA., Sahenk Z; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA.; Department of Neurology, The Ohio State University College of Medicine, Columbus, OH, USA., Rodino-Klapac LR; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA., Harper SQ; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA.
Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2017 Dec 24; Vol. 8, pp. 121-130. Date of Electronic Publication: 2017 Dec 24 (Print Publication: 2018).
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
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  Data: Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD.
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  Data: <searchLink fieldCode="AU" term="%22Wallace+LM%22">Wallace LM</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, 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, OH, 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, OH, 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, OH, 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, OH, 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, OH, USA.<br /><searchLink fieldCode="AU" term="%22Griffin+DA%22">Griffin DA</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.<br /><searchLink fieldCode="AU" term="%22Herman+AC%22">Herman AC</searchLink>; Research Information Solutions and Innovation Infrastructure, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.<br /><searchLink fieldCode="AU" term="%22Sahenk+Z%22">Sahenk Z</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA.; Department of Neurology, The Ohio State University College of Medicine, Columbus, OH, 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, OH, USA.; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA.<br /><searchLink fieldCode="AU" term="%22Harper+SQ%22">Harper SQ</searchLink>; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA.
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  Data: <searchLink fieldCode="JN" term="%22101624857%22">Molecular therapy. Methods & clinical development</searchLink> [Mol Ther Methods Clin Dev] 2017 Dec 24; Vol. 8, pp. 121-130. <i>Date of Electronic Publication: </i>2017 Dec 24 (<i>Print Publication: </i>2018).
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Cell+Press%22">Cell Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>101624857 <i>Publication Model: </i>eCollection <i>Cited Medium: </i>Print <i>ISSN: </i>2329-0501 (Print) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2223290501%22">23290501 </searchLink><i>NLM ISO Abbreviation: </i>Mol Ther Methods Clin Dev <i>Subsets: </i>PubMed not MEDLINE
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        Value: 10.1016/j.omtm.2017.12.005
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              Text: 2017 Dec 24
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