In vivo genome editing of the albumin locus as a platform for protein replacement therapy (Q36143241)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26297739%20AND%20SRC:MED&resulttype=core&format=json

22 February 2020

title

In vivo genome editing of the albumin locus as a platform for protein replacement therapy (English)

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22 February 2020

1

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22 February 2020

Xavier M Anguela

2

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22 February 2020

Yannick Doyon

3

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22 February 2020

Philip D Gregory

13

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22 February 2020

Katherine A. High

16

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22 February 2020

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Edward J Rebar

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22 February 2020

7

David E Paschon

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22 February 2020

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David Shivak

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22 February 2020

author name string

Thomas Wechsler

4

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22 February 2020

Russell C DeKelver

5

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22 February 2020

Scott Sproul

6

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22 February 2020

Jeffrey C Miller

8

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22 February 2020

Robert J Davidson

9

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22 February 2020

Shangzhen Zhou

11

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22 February 2020

Julianne Rieders

12

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22 February 2020

Michael C Holmes

14

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22 February 2020

language of work or name

English

0 references

publication date

21 August 2015

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22 February 2020

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22 February 2020

volume

126

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22 February 2020

issue

15

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22 February 2020

page(s)

1777-1784

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Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

22 February 2020

cites work

1 reference

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Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

2 October 2017

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Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.

2 October 2017

1 reference

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Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV

2 October 2017

1 reference

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Robust ZFN-mediated genome editing in adult hemophilic mice.

2 October 2017

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Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

2 October 2017

1 reference

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Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype

2 October 2017

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AAV2 gene therapy readministration in three adults with congenital blindness.

2 October 2017

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Adenovirus-associated virus vector-mediated gene transfer in hemophilia B

2 October 2017

1 reference

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Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector.

2 October 2017

1 reference

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Revealing off-target cleavage specificities of zinc-finger nucleases by in vitro selection

2 October 2017

1 reference

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

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4600017

Vector characterization methods for quality control testing of recombinant adeno-associated viruses

2 October 2017

1 reference

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Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures

2 October 2017

1 reference

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

2 October 2017

1 reference

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Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

2 October 2017

1 reference

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Efficient gene targeting mediated by adeno-associated virus and DNA double-strand breaks

2 October 2017

1 reference

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Analysis of Relative Gene Expression Data Using Real-Time Quantitative PCR and the 2−ΔΔCT Method

2 October 2017

1 reference

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2 October 2017

1 reference

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Targeted genome editing in human repopulating haematopoietic stem cells

2 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4600017

Overcoming preexisting humoral immunity to AAV using capsid decoys

2 July 2018

1 reference

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Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease

2 July 2018

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

2 July 2018

1 reference

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Engineering a novel secretion signal for cross-host recombinant protein expression

23 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4600017

Hurler and Hunter syndromes: mutual correction of the defect in cultured fibroblasts

23 September 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/26297739

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Novel forms of B-domain-deleted recombinant factor VIII molecules. Construction and biochemical characterization

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/26297739

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1182/BLOOD-2014-12-615492

1 reference

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22 February 2020

1 reference

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22 February 2020

1 reference