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

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Bibliographic Details
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
Description
ISSN:1525-0024
DOI:10.1016/j.ymthe.2020.04.007