首页> Human gene therapy> Adeno-Associated Virus-Mediated Gene Therapy in the Mashlool, Atp1a3Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood.

Adeno-Associated Virus-Mediated Gene Therapy in the Mashlool, Atp1a3Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood.

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第一作者： Arsen S,Hunanyan

第一单位： Division of Pediatric Neurology and Developmental Medicine, Department of Pediatrics, Duke University, Durham, North Carolina, USA.

作者： Arsen S,Hunanyan ^[1] ; Boris,Kantor ^[2] ; Ram S,Puranam ^[3] ; Courtney,Elliott ^[1] ; Angela,McCall ^[4] ; Justin,Dhindsa ^[4] ; Promila,Pagadala ^[5] ; Keri,Wallace ^[1] ; Jordan,Poe ^[2] ; Talha,Gunduz ^[1] ; Aravind,Asokan ^[6] ; Dwight D,Koeberl ^[7] ; Mai K,ElMallah ^[4] ; Mohamad A,Mikati ^[8]

作者单位： Division of Pediatric Neurology and Developmental Medicine, Department of Pediatrics, Duke University, Durham, North Carolina, USA. ^[1] Viral Vector Core, Department of Neurobiology, Duke University, Durham, North Carolina, USA. ^[2] Department of Neurobiology, Duke University, Durham, North Carolina, USA. ^[3] Division of Pediatric Pulmonary Medicine, Department of Pediatrics, Duke University, Durham, North Carolina, USA. ^[4] Department of Clinical and Translational Science Institute, Duke University, Durham, North Carolina, USA. ^[5] Department of Surgery, Duke University, Durham, North Carolina, USA.;Molecular Genetics and Microbiology, Duke University, Durham, North Carolina, USA. ^[6] Division of Medical Genetics, Department of Pediatrics, Duke University, Durham, North Carolina, USA. ^[7] Division of Pediatric Neurology and Developmental Medicine, Department of Pediatrics, Duke University, Durham, North Carolina, USA.;Department of Neurobiology, Duke University, Durham, North Carolina, USA. ^[8]

关键词 AAV9 Mashl+/? mice Na+/K+-ATPase alternating hemiplegia of childhood

医学主题词动物(Animals);依赖病毒(Dependovirus);偏瘫(Hemiplegia);小鼠(Mice);突变(Mutation);钠钾交换ATP酶(Sodium-Potassium-Exchanging ATPase)

DOI 10.1089/hum.2020.191

PMID 33577387

发布时间 2022-05-31

基金项目

R01 HD099486/HD/NICHD NIH HHS/United States UL1 TR002553/TR/NCATS NIH HHS/United States

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