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RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model.

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Title: RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model.
Authors: Aimiuwu OV; Institute for Genomic Medicine and Department of Genetics and Development, Columbia University Irving Medical Center, New York, NY 10032, USA., Fowler AM; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., Sah M; Institute for Genomic Medicine and Department of Genetics and Development, Columbia University Irving Medical Center, New York, NY 10032, USA., Teoh JJ; Institute for Genomic Medicine and Department of Genetics and Development, Columbia University Irving Medical Center, New York, NY 10032, USA., Kanber A; Institute for Genomic Medicine and Department of Genetics and Development, Columbia University Irving Medical Center, New York, NY 10032, USA., Pyne NK; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., Petri S; Institute for Genomic Medicine and Department of Genetics and Development, Columbia University Irving Medical Center, New York, NY 10032, USA., Rosenthal-Weiss C; Institute for Genomic Medicine and Department of Genetics and Development, Columbia University Irving Medical Center, New York, NY 10032, USA., Yang M; Institute for Genomic Medicine and Department of Genetics and Development, Columbia University Irving Medical Center, New York, NY 10032, USA., Harper SQ; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH 43205, USA., Frankel WN; Institute for Genomic Medicine and Department of Genetics and Development, Columbia University Irving Medical Center, New York, NY 10032, USA. Electronic address: wf2218@cumc.columbia.edu.
Source: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2020 Jul 08; Vol. 28 (7), pp. 1706-1716. Date of Electronic Publication: 2020 Apr 16.
Publication Type: Journal Article; Research Support, N.I.H., Extramural
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model.
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  Data: <searchLink fieldCode="AU" term="%22Aimiuwu+OV%22">Aimiuwu OV</searchLink>; Institute for Genomic Medicine and Department of Genetics and Development, Columbia University Irving Medical Center, New York, NY 10032, USA.<br /><searchLink fieldCode="AU" term="%22Fowler+AM%22">Fowler AM</searchLink>; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.<br /><searchLink fieldCode="AU" term="%22Sah+M%22">Sah M</searchLink>; Institute for Genomic Medicine and Department of Genetics and Development, Columbia University Irving Medical Center, New York, NY 10032, USA.<br /><searchLink fieldCode="AU" term="%22Teoh+JJ%22">Teoh JJ</searchLink>; Institute for Genomic Medicine and Department of Genetics and Development, Columbia University Irving Medical Center, New York, NY 10032, USA.<br /><searchLink fieldCode="AU" term="%22Kanber+A%22">Kanber A</searchLink>; Institute for Genomic Medicine and Department of Genetics and Development, Columbia University Irving Medical Center, New York, NY 10032, USA.<br /><searchLink fieldCode="AU" term="%22Pyne+NK%22">Pyne NK</searchLink>; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.<br /><searchLink fieldCode="AU" term="%22Petri+S%22">Petri S</searchLink>; Institute for Genomic Medicine and Department of Genetics and Development, Columbia University Irving Medical Center, New York, NY 10032, USA.<br /><searchLink fieldCode="AU" term="%22Rosenthal-Weiss+C%22">Rosenthal-Weiss C</searchLink>; Institute for Genomic Medicine and Department of Genetics and Development, Columbia University Irving Medical Center, New York, NY 10032, USA.<br /><searchLink fieldCode="AU" term="%22Yang+M%22">Yang M</searchLink>; Institute for Genomic Medicine and Department of Genetics and Development, Columbia University Irving Medical Center, New York, NY 10032, USA.<br /><searchLink fieldCode="AU" term="%22Harper+SQ%22">Harper SQ</searchLink>; Center for Gene Therapy, The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH 43205, USA.<br /><searchLink fieldCode="AU" term="%22Frankel+WN%22">Frankel WN</searchLink>; Institute for Genomic Medicine and Department of Genetics and Development, Columbia University Irving Medical Center, New York, NY 10032, USA. Electronic address: wf2218@cumc.columbia.edu.
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  Data: <searchLink fieldCode="JN" term="%22100890581%22">Molecular therapy : the journal of the American Society of Gene Therapy</searchLink> [Mol Ther] 2020 Jul 08; Vol. 28 (7), pp. 1706-1716. <i>Date of Electronic Publication: </i>2020 Apr 16.
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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>100890581 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1525-0024 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2215250016%22">15250016 </searchLink><i>NLM ISO Abbreviation: </i>Mol Ther <i>Subsets: </i>MEDLINE
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        Value: 10.1016/j.ymthe.2020.04.007
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              Text: 2020 Jul 08
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