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

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

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2018 nî lūn-bûn

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2018年の論文

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2018年学术文章

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2018年学术文章

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2018年学术文章

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2018年学术文章

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2018年学术文章

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2018年学术文章

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2018年學術文章

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2018年學術文章

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name

Targeted in vivo knock-in of h ...... viral delivery of CRISPR/Cas9.

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Targeted in vivo knock-in of h ...... viral delivery of CRISPR/Cas9.

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type

Item

label

Targeted in vivo knock-in of h ...... viral delivery of CRISPR/Cas9.

@en

Targeted in vivo knock-in of h ...... viral delivery of CRISPR/Cas9.

@nl

prefLabel

Targeted in vivo knock-in of h ...... viral delivery of CRISPR/Cas9.

@en

Targeted in vivo knock-in of h ...... viral delivery of CRISPR/Cas9.

@nl

P1476

Targeted in vivo knock-in of h ...... viral delivery of CRISPR/Cas9.

@en

P2093

Calvin J Stephens

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Elena Kashentseva

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Lyudmila Kaliberova

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William Everett

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P2860

Adeno-associated virus vector integration

In vivo genome editing restores haemostasis in a mouse model of haemophilia

Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo

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

Unexpected mutations after CRISPR–Cas9 editing in vivo

Gene therapy for Wiskott-Aldrich syndrome--long-term efficacy and genotoxicity.

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

A conditional knockout mouse model reveals endothelial cells as the principal and possibly exclusive source of plasma factor VIII.

CRISPR/Cas9 targeting events cause complex deletions and insertions at 17 sites in the mouse genome.

A comparison of exogenous promoter activity at the ROSA26 locus using a ΦiC31 integrase mediated cassette exchange approach in mouse ES cells

P2888

s41434-018-0003-1

P304

139-156

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P31

scholarly article

P356

10.1038/S41434-018-0003-1

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P433

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P478

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P50

David T. Curiel

P577

2018-03-27T00:00:00Z

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P6179

1101781361

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P698

29588497

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P921

Adenoviridae

CRISPR

P932

5919923

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