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Biodistribution of AAV1, AAV5, AAV9, and AAVDJ serotypes after intra-cisterna magna delivery in non-human primates.

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第一作者： Takuro,Okai

第一单位： Neuroscience Drug Discovery Unit, Research, Takeda Pharmaceutical Company Limited, Fujisawa 251-8555, Japan.

作者： Takuro,Okai ^[1] ; Sho,Sato ^[2] ; Hironobu,Yasuno ^[3] ; Miyu,Nakayama ^[2] ; Syunsuke,Yamamoto ^[2] ; Sebastian,Sjöqvist ^[1] ; Kentaro,Otake ^[1] ; Masato,Nakashima ^[1] ; Mugdha,Deshpande ^[4] ; Elizabeth,Galbreath ^[3] ; Jeong-Ho,Oak ^[3] ; Saku,Miyamoto ^[3] ; Gabriele,Proetzel ^[5]

作者单位： Neuroscience Drug Discovery Unit, Research, Takeda Pharmaceutical Company Limited, Fujisawa 251-8555, Japan. ^[1] Center of Excellence for Drug Metabolism, Pharmacokinetics and Modeling, Preclinical and Translational Sciences, Research, Takeda Pharmaceutical Company Limited, Fujisawa 251-8555, Japan. ^[2] Drug Safety Research and Evaluation, Preclinical and Translational Sciences, Research, Takeda Pharmaceutical Company Limited, Fujisawa 251-8555, Japan. ^[3] Takeda Development Center Americas, Cambridge, MA 02142, USA. ^[4] Neuroscience Drug Discovery Unit, Research, Takeda Development Center Americas, Inc, Cambridge, MA 02142, USA. ^[5]

关键词 AAV CNS GBA1 Glucosylceramidase beta 1 NHP biodistribution dorsal root ganglion gene therapy intra-cisterna magna non-human primate

DOI 10.1016/j.omtm.2025.101593

PMID 41078870

发布时间 2025-10-13

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Molecular therapy. Methods & clinical development

2025年33卷4期

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Molecular therapy. Methods & clinical development

2025年33卷4期

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Biodistribution of AAV1, AAV5, AAV9, and AAVDJ serotypes after intra-cisterna magna delivery in non-human primates.

Molecular therapy. Methods & clinical development

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Biodistribution of AAV1, AAV5, AAV9, and AAVDJ serotypes after intra-cisterna magna delivery in non-human primates.

Molecular therapy. Methods & clinical development

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Molecular therapy. Methods & clinical development

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