Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.-论文-万方医学网

第一作者： Kathrin,Meyer

第一单位： The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA.

作者： Kathrin,Meyer ^[1] ; Laura,Ferraiuolo ^[1] ; Leah,Schmelzer ^[1] ; Lyndsey,Braun ^[1] ; Vicki,McGovern ^[2] ; Shibi,Likhite ^[3] ; Olivia,Michels ^[1] ; Alessandra,Govoni ^[1] ; Julie,Fitzgerald ^[4] ; Pablo,Morales ^[5] ; Kevin D,Foust ^[4] ; Jerry R,Mendell ^[6] ; Arthur H M,Burghes ^[2] ; Brian K,Kaspar ^[6]

作者单位： The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA. ^[1] Department of Molecular & Cellular Biochemistry, The Ohio State University Medical Center, Columbus, Ohio, USA. ^[2] 1] The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA [2] Molecular, Cellular & Developmental Biology Graduate Program, The Ohio State University, Columbus, Ohio, USA. ^[3] Department of Neuroscience, The Ohio State University, Columbus, Ohio, USA. ^[4] Mannheimer Foundation, Inc., Homestead, Florida, USA. ^[5] 1] The Research Institute at Nationwide Children's Hospital, Columbus, Ohio, USA [2] Molecular, Cellular & Developmental Biology Graduate Program, The Ohio State University, Columbus, Ohio, USA [3] Department of Neuroscience, The Ohio State University, Columbus, Ohio, USA. ^[6]

医学主题词动物(Animals);动物, 新生(Animals, Newborn);脑干(Brain Stem);大脑皮质(Cerebral Cortex);DNA, 互补(DNA, Complementary);依赖病毒(Dependovirus);疾病模型, 动物(Disease Models, Animal);剂量效应关系, 药物(Dose-Response Relationship, Drug);基因表达(Gene Expression);遗传载体(Genetic Vectors);注射, 硬膜外(Injections, Epidural);成束猴(Macaca fascicularis);小鼠(Mice);小鼠, 基因敲除(Mice, Knockout);运动神经元(Motor Neurons);肌萎缩, 脊髓性(Muscular Atrophy, Spinal);脊髓(Spinal Cord);运动神经元生存蛋白质1(Survival of Motor Neuron 1 Protein);转导, 遗传(Transduction, Genetic);转基因(Transgenes)

DOI 10.1038/mt.2014.210

PMID 25358252

发布时间 2021-10-21

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Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.

Molecular therapy

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Molecular therapy

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Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.

Molecular therapy

相似文献

同项目论文

Molecular therapy

相关期刊

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