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Antisense oligonucleotide therapy for KCNT1 encephalopathy.
Burbano, Lisseth Estefania; Li, Melody; Jancovski, Nikola; Jafar-Nejad, Paymaan; Richards, Kay; Sedo, Alicia; Soriano, Armand; Rollo, Ben; Jia, Linghan; Gazina, Elena V; Piltz, Sandra; Adikusuma, Fatwa; Thomas, Paul Q; Kopsidas, Helen; Rigo, Frank; Reid, Christopher A; Maljevic, Snezana; Petrou, Steven.
Afiliação
  • Burbano LE; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria, Australia.
  • Li M; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria, Australia.
  • Jancovski N; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria, Australia.
  • Jafar-Nejad P; Ionis Pharmaceuticals, Carlsbad, California, USA.
  • Richards K; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria, Australia.
  • Sedo A; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria, Australia.
  • Soriano A; Ionis Pharmaceuticals, Carlsbad, California, USA.
  • Rollo B; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria, Australia.
  • Jia L; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria, Australia.
  • Gazina EV; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria, Australia.
  • Piltz S; School of Medicine, University of Adelaide, Adelaide, South Australia, Australia.
  • Adikusuma F; School of Medicine, University of Adelaide, Adelaide, South Australia, Australia.
  • Thomas PQ; School of Medicine, University of Adelaide, Adelaide, South Australia, Australia.
  • Kopsidas H; South Australian Health and Medical Research Institute, Adelaide, South Australia, Australia.
  • Rigo F; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria, Australia.
  • Reid CA; Ionis Pharmaceuticals, Carlsbad, California, USA.
  • Maljevic S; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria, Australia.
  • Petrou S; The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria, Australia.
JCI Insight ; 7(23)2022 12 08.
Article em En | MEDLINE | ID: mdl-36173683

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Encefalopatias Limite: Animals Idioma: En Ano de publicação: 2022 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Encefalopatias Limite: Animals Idioma: En Ano de publicação: 2022 Tipo de documento: Article