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Precision Antisense Oligonucleotide Therapy Amenability for Infantile Genetic Epilepsies.

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Title: Precision Antisense Oligonucleotide Therapy Amenability for Infantile Genetic Epilepsies.
Authors: Sherrill E; Division of Genetics and Genomics, Department of Pediatrics, Boston Children's Hospital, Boston, Massachusetts., Cheerie D; Program in Genetics and Genome Biology, SickKids Research Institute, Toronto, Ontario, Canada., Beck CJ; Murdoch Children's Research Institute, Parkville, Victoria, Australia., Whittle EF; Genetics and Genomic Medicine, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, United Kingdom.; UK Platform for Nucleic Acid Therapies (UPNAT), London, United Kingdom., Shafi Y; Genetics and Genomic Medicine, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, United Kingdom.; UK Platform for Nucleic Acid Therapies (UPNAT), London, United Kingdom., Chandler NJ; North Thames Genomic Laboratory Hub, Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom., Christodoulou J; Murdoch Children's Research Institute, Parkville, Victoria, Australia.; Department of Paediatrics, University of Melbourne, Melbourne, Victoria, Australia., Daniel J; Department of Genetics, Evolution and Environment (GEE), University College London, London, United Kingdom.; UCL Great Ormond Street Institute of Child Health (ICH), London, United Kingdom., Hassell J; Department of Neurology, Great Ormond Street Hospital, London, United Kingdom., Lachgar-Ruiz M; North Thames Genomic Laboratory Hub, Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom., Mulhern S; Murdoch Children's Research Institute, Parkville, Victoria, Australia.; Victorian Clinical Genetics Services, Melbourne, Victoria, Australia., Scotchman E; North Thames Genomic Laboratory Hub, Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom., Sidhu J; Program in Genetics and Genome Biology, SickKids Research Institute, Toronto, Ontario, Canada., Tedja CF; Division of Biosciences, University College London, London, United Kingdom., Chitty LS; Genetics and Genomic Medicine, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, United Kingdom.; North Thames Genomic Laboratory Hub, Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom., Cross JH; Department of Neurology, Great Ormond Street Hospital, London, United Kingdom.; Developmental Neurosciences, UCL Great Ormond Street Institute of Child Health, London, United Kingdom., Scheffer IE; Murdoch Children's Research Institute, Parkville, Victoria, Australia.; Department of Paediatrics, University of Melbourne, Melbourne, Victoria, Australia.; Department of Medicine, University of Melbourne, Melbourne, Victoria, Australia.; Austin Health, and Florey Institute of Neuroscience and Mental Health, Melbourne, Victoria, Australia., Zhou H; Genetics and Genomic Medicine, UCL Great Ormond Street Institute of Child Health, London WC1N 1EH, United Kingdom.; UK Platform for Nucleic Acid Therapies (UPNAT), London, United Kingdom., Yu TW; Division of Genetics and Genomics, Department of Pediatrics, Boston Children's Hospital, Boston, Massachusetts.; Department of Pediatrics, Harvard Medical School, Boston, Massachusetts.; F.M. Kirby Neurobiology Center, Boston Children's Hospital, Boston, Massachusetts., Chau V; Division of Neurology, Hospital for Sick Children, Toronto, Ontario, Canada.; Neuroscience and Mental Health Program, SickKids Research Institute, Toronto, Ontario, Canada.; Department of Paediatrics, Temerty Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada., Stephenson SEM; Murdoch Children's Research Institute, Parkville, Victoria, Australia.; Department of Paediatrics, University of Melbourne, Melbourne, Victoria, Australia., Poduri A; F.M. Kirby Neurobiology Center, Boston Children's Hospital, Boston, Massachusetts.; Department of Neurology, Boston Children's Hospital, Boston, Massachusetts.; Department of Neurology, Harvard Medical School, Boston, Massachusetts.; Children's Rare Disease Collaborative, Boston Children's Hospital, Boston, Massachusetts., Howell KB; Murdoch Children's Research Institute, Parkville, Victoria, Australia.; Department of Paediatrics, University of Melbourne, Melbourne, Victoria, Australia.; Department of Neurology, Royal Children's Hospital, Melbourne, Victoria, Australia., McTague A; Department of Neurology, Great Ormond Street Hospital, London, United Kingdom.; Developmental Neurosciences, UCL Great Ormond Street Institute of Child Health, London, United Kingdom., Costain G; Program in Genetics and Genome Biology, SickKids Research Institute, Toronto, Ontario, Canada.; Division of Clinical & Metabolic Genetics, Hospital for Sick Children, Toronto, Ontario, Canada.; Department of Paediatrics, Temerty Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada.; Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada., D'Gama AM; Department of Pediatrics, Harvard Medical School, Boston, Massachusetts.; F.M. Kirby Neurobiology Center, Boston Children's Hospital, Boston, Massachusetts.; Children's Rare Disease Collaborative, Boston Children's Hospital, Boston, Massachusetts.; Division of Newborn Medicine, Department of Pediatrics, Boston Children's Hospital, Boston, Massachusetts.
Corporate Authors: Gene-STEPS Study Group
Source: JAMA neurology [JAMA Neurol] 2026 Jun 01; Vol. 83 (6), pp. 598-601.
Publication Type: Journal Article
Journal Info: Publisher: American Medical Association Country of Publication: United States NLM ID: 101589536 Publication Model: Print Cited Medium: Internet ISSN: 2168-6157 (Electronic) Linking ISSN: 21686149 NLM ISO Abbreviation: JAMA Neurol Subsets: MEDLINE; In Process
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  Data: Precision Antisense Oligonucleotide Therapy Amenability for Infantile Genetic Epilepsies.
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Kirby Neurobiology Center, Boston Children's Hospital, Boston, Massachusetts.<br /><searchLink fieldCode="AU" term="%22Chau+V%22">Chau V</searchLink>; Division of Neurology, Hospital for Sick Children, Toronto, Ontario, Canada.; Neuroscience and Mental Health Program, SickKids Research Institute, Toronto, Ontario, Canada.; Department of Paediatrics, Temerty Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada.<br /><searchLink fieldCode="AU" term="%22Stephenson+SEM%22">Stephenson SEM</searchLink>; Murdoch Children's Research Institute, Parkville, Victoria, Australia.; Department of Paediatrics, University of Melbourne, Melbourne, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22Poduri+A%22">Poduri A</searchLink>; F.M. Kirby Neurobiology Center, Boston Children's Hospital, Boston, Massachusetts.; Department of Neurology, Boston Children's Hospital, Boston, Massachusetts.; Department of Neurology, Harvard Medical School, Boston, Massachusetts.; Children's Rare Disease Collaborative, Boston Children's Hospital, Boston, Massachusetts.<br /><searchLink fieldCode="AU" term="%22Howell+KB%22">Howell KB</searchLink>; Murdoch Children's Research Institute, Parkville, Victoria, Australia.; Department of Paediatrics, University of Melbourne, Melbourne, Victoria, Australia.; Department of Neurology, Royal Children's Hospital, Melbourne, Victoria, Australia.<br /><searchLink fieldCode="AU" term="%22McTague+A%22">McTague A</searchLink>; Department of Neurology, Great Ormond Street Hospital, London, United Kingdom.; Developmental Neurosciences, UCL Great Ormond Street Institute of Child Health, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Costain+G%22">Costain G</searchLink>; Program in Genetics and Genome Biology, SickKids Research Institute, Toronto, Ontario, Canada.; Division of Clinical & Metabolic Genetics, Hospital for Sick Children, Toronto, Ontario, Canada.; Department of Paediatrics, Temerty Faculty of Medicine, University of Toronto, Toronto, Ontario, Canada.; Department of Molecular Genetics, University of Toronto, Toronto, Ontario, Canada.<br /><searchLink fieldCode="AU" term="%22D'Gama+AM%22">D'Gama AM</searchLink>; Department of Pediatrics, Harvard Medical School, Boston, Massachusetts.; F.M. Kirby Neurobiology Center, Boston Children's Hospital, Boston, Massachusetts.; Children's Rare Disease Collaborative, Boston Children's Hospital, Boston, Massachusetts.; Division of Newborn Medicine, Department of Pediatrics, Boston Children's Hospital, Boston, Massachusetts.
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