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

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Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR/Cas9.

P2860

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

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http://www.wikidata.org/entity/Q40075764

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2017 nî lūn-bûn

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2017年の論文

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2017年学术文章

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2017年学术文章

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2017年学术文章

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2017年學術文章

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2017年學術文章

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Neonatal Gene Therapy for Hemo ...... aky Expression of Viral Genes.

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type

Item

label

Neonatal Gene Therapy for Hemo ...... aky Expression of Viral Genes.

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prefLabel

Neonatal Gene Therapy for Hemo ...... aky Expression of Viral Genes.

@en

P1476

Neonatal Gene Therapy for Hemo ...... aky Expression of Viral Genes.

@en

P2093

Fuminori Sakurai

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Kazuo Ohashi

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Masashi Tachibana

http://www.w3.org/2001/XMLSchema#string

Shunsuke Iizuka

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P2860

Safety and efficacy of gene transfer for Leber's congenital amaurosis

Identification of tissue-specific microRNAs from mouse

Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer

Gutless adenovirus: last-generation adenovirus for gene therapy

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency.

Transient pretreatment with glucocorticoid ablates innate toxicity of systemically delivered adenoviral vectors without reducing efficacy.

Gene therapy for the hemophilias.

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial.

Neonatal helper-dependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII

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183-193

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scholarly article

P356

10.1016/J.OMTM.2017.07.001

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P478

http://www.w3.org/2001/XMLSchema#string

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Hiroyuki Mizuguchi

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2017-07-08T00:00:00Z

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28828393

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P932

5552065

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

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P2860

P2860

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

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type

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P1433

P1476

P2093

P2860

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P356

P478

P50

P577

P698

P921

P932

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P1433

P1476

P2093

P2860

P304

P31

P356

P478

P50

P577

P698

P921

P932