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RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model.
Aimiuwu, Osasumwen V; Fowler, Allison M; Sah, Megha; Teoh, Jia Jie; Kanber, Ayla; Pyne, Nettie K; Petri, Sabrina; Rosenthal-Weiss, Chana; Yang, Mu; Harper, Scott Q; Frankel, Wayne N.
Affiliation
  • 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.
Mol Ther ; 28(7): 1706-1716, 2020 07 08.
Article in En | MEDLINE | ID: mdl-32353324
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Dynamin I / MicroRNAs / Epileptic Syndromes Limits: Animals / Humans Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2020 Document type: Article Affiliation country: Estados Unidos Country of publication: Estados Unidos
Fulltext
Add to My VHL
Print
XML
PubMed Links
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Dynamin I / MicroRNAs / Epileptic Syndromes Limits: Animals / Humans Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2020 Document type: Article Affiliation country: Estados Unidos Country of publication: Estados Unidos