In vivo genome editing restores haemostasis in a mouse model of haemophilia (Q28241725)

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6 February 2020

title

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

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6 February 2020

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6 February 2020

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6 February 2020

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6 February 2020

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6 February 2020

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6 February 2020

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6 February 2020

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6 February 2020

Edward J Rebar

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6 February 2020

David E Paschon

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6 February 2020

Hojun Li

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6 February 2020

Tianjian Li

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6 February 2020

Sunnie Y Wong

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6 February 2020

Anand S Bhagwat

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6 February 2020

Nirav Malani

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6 February 2020

Lacramiora Ivanciu

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6 February 2020

Samuel L Murphy

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6 February 2020

Jonathan D Finn

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6 February 2020

Fayaz R Khazi

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6 February 2020

Shangzhen Zhou

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6 February 2020

Michael C Holmes

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6 February 2020

language of work or name

English

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publication date

26 June 2011

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6 February 2020

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6 February 2020

volume

475

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6 February 2020

issue

7355

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6 February 2020

page(s)

217-221

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Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

6 February 2020

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3152293

Genome editing with engineered zinc finger nucleases

20 March 2017

1 reference

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Heritable targeted gene disruption in zebrafish using designed zinc-finger nucleases

20 March 2017

1 reference

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Effect of gene therapy on visual function in Leber's congenital amaurosis

20 March 2017

1 reference

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

20 March 2017

1 reference

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Highly efficient endogenous human gene correction using designed zinc-finger nucleases

20 March 2017

1 reference

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LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

20 March 2017

1 reference

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Selection of target sites for mobile DNA integration in the human genome

20 March 2017

1 reference

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Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases

20 March 2017

1 reference

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Chimeric nucleases stimulate gene targeting in human cells

7 April 2017

1 reference

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Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3152293

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

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3152293

Assessing the potential for AAV vector genotoxicity in a murine model

7 April 2017

1 reference

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Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3152293

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

29 September 2017

1 reference

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Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3152293

Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3152293

Adeno-associated virus vectors integrate at chromosome breakage sites

29 September 2017

1 reference

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AAV serotype 2 vectors preferentially integrate into active genes in mice

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3152293

Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3152293

Stimulation of homologous recombination through targeted cleavage by chimeric nucleases

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3152293

Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3152293

Disruption of overlapping transcripts in the ROSA beta geo 26 gene trap strain leads to widespread expression of beta-galactosidase in mouse embryos and hematopoietic cells

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3152293

A coagulation factor IX-deficient mouse model for human hemophilia B.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3152293

Germ line origins of de novo mutations in hemophilia B families

2 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3152293

High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency

2 June 2018

1 reference

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Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

27 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3152293

A reproducible and well-tolerated method for 2/3 partial hepatectomy in mice

27 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3152293

27 November 2018

Identifiers

DOI

10.1038/NATURE10177

2 references

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Dimensions Publication ID

6 February 2020

1041862601

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Fatcat ID

release_6kidcgq56naefaymkcuux4fpoi

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Fatcat

https://api.fatcat.wiki/v0/release/6kidcgq56naefaymkcuux4fpoi

mapped directly with Wikidata item

24 November 2022

based on heuristic

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2 references

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6 February 2020

PubMed publication ID

21706032

2 references

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