Antisense oligonucleotide therapy for KCNT1 encephalopathy.

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第一作者： Lisseth Estefania,Burbano

第一单位： The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria, Australia.

作者： Lisseth Estefania,Burbano ^[1] ; Melody,Li ^[1] ; Nikola,Jancovski ^[1] ; Paymaan,Jafar-Nejad ^[2] ; Kay,Richards ^[1] ; Alicia,Sedo ^[1] ; Armand,Soriano ^[2] ; Ben,Rollo ^[1] ; Linghan,Jia ^[1] ; Elena V,Gazina ^[1] ; Sandra,Piltz ^[3] ; Fatwa,Adikusuma ^[3] ; Paul Q,Thomas ^[3] ; Helen,Kopsidas ^[4] ; Frank,Rigo ^[1] ; Christopher A,Reid ^[2] ; Snezana,Maljevic ^[1] ; Steven,Petrou ^[1]

作者单位： The Florey Institute of Neuroscience and Mental Health, The University of Melbourne, Parkville, Victoria, Australia. ^[1] Ionis Pharmaceuticals, Carlsbad, California, USA. ^[2] School of Medicine, University of Adelaide, Adelaide, South Australia, Australia. ^[3] South Australian Health and Medical Research Institute, Adelaide, South Australia, Australia. ^[4] Praxis Precision Medicines, Cambridge, Massachusetts, USA. ^[5]

关键词 Epilepsy Gene therapy Genetics Mouse models Neuroscience

主题词小鼠(Mice);动物(Animals);脑疾病(Brain Diseases);发作(Seizures)

DOI 10.1172/jci.insight.146090

PMID 36173683

发布时间 2023-01-11

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2022年7卷23期

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Antisense oligonucleotide therapy for KCNT1 encephalopathy.

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Antisense oligonucleotide therapy for KCNT1 encephalopathy.

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