Neonatal Gene Therapy for Hemophilia B by a Novel Adenovirus Vector Showing Reduced Leaky Expression of Viral Genes.

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第一作者： Shunsuke,Iizuka

第一单位： Laboratory of Biochemistry and Molecular Biology, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka 565-0871, Japan.

作者： Shunsuke,Iizuka ^[1] ; Fuminori,Sakurai ^[2] ; Masashi,Tachibana ^[3] ; Kazuo,Ohashi ^[1] ; Hiroyuki,Mizuguchi ^[4]

作者单位： Laboratory of Biochemistry and Molecular Biology, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka 565-0871, Japan. ^[1] Laboratory of Biochemistry and Molecular Biology, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka 565-0871, Japan.;Clinical Drug Development Unit, Laboratory of Regulatory Sciences for Oligonucleotide Therapeutics, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka 565-0871, Japan. ^[2] Laboratory of Biochemistry and Molecular Biology, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka 565-0871, Japan.;Laboratory of Biopharmaceutics, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka 565-0871, Japan. ^[3] Laboratory of Biochemistry and Molecular Biology, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka 565-0871, Japan.;iPS Cell-Based Research Project on Hepatic Toxicity and Metabolism, Graduate School of Pharmaceutical Sciences, Osaka University, Osaka 565-0871, Japan.;The Center for Advanced Medical Engineering and Informatics, Osaka University, Osaka 565-0871, Japan.;Laboratory of Hepatocyte Regulation, National Institute of Biomedical Innovation, Health and Nutrition, Osaka 567-0085, Japan.;Global Center for Advanced Medical Engineering and Informatics, Osaka University, Osaka 565-0871, Japan. ^[4]

关键词 adenovirus vector gene therapy hemophilia B

DOI 10.1016/j.omtm.2017.07.001

PMID 28828393

发布时间 2020-09-30

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Molecular therapy. Methods & clinical development

2017年6卷

183-193页

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Neonatal Gene Therapy for Hemophilia B by a Novel Adenovirus Vector Showing Reduced Leaky Expression of Viral Genes.

Molecular therapy. Methods & clinical development

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Molecular therapy. Methods & clinical development

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Neonatal Gene Therapy for Hemophilia B by a Novel Adenovirus Vector Showing Reduced Leaky Expression of Viral Genes.

Molecular therapy. Methods & clinical development

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Molecular therapy. Methods & clinical development

相关期刊

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