(Q36951279)

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In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA.

scientific article published on 11 February 2016

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scholarly article

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Europe PubMed Central

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26865405%20AND%20SRC:MED&resulttype=core&format=json

7 March 2020

In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA (English)

1 reference

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26865405%20AND%20SRC:MED&resulttype=core&format=json

7 March 2020

glycogen storage disease

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based on heuristic

inferred from title

Elizabeth D Brooks

2

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https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:26865405%20AND%20SRC:MED&resulttype=core&format=json

7 March 2020

Pablo Perez-Pinera

3

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7 March 2020

author name string

Dustin J Landau

1

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7 March 2020

Hiruni Amarasekara

4

1 reference

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7 March 2020

Adam Mefferd

5

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7 March 2020

Songtao Li

6

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7 March 2020

Andrew Bird

7

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7 March 2020

Charles A Gersbach

8

1 reference

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7 March 2020

Dwight D Koeberl

9

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7 March 2020

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11 February 2016

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7 March 2020

Molecular Therapy

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7 March 2020

24

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7 March 2020

4

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7 March 2020

697-706

1 reference

Europe PubMed Central

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7 March 2020

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

1 reference

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9 September 2017

Diagnosis and management of glycogen storage disease type I: a practice guideline of the American College of Medical Genetics and Genomics

1 reference

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9 September 2017

Robust ZFN-mediated genome editing in adult hemophilic mice.

1 reference

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9 September 2017

Advances in targeted genome editing

1 reference

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9 September 2017

Prevention of hepatocellular adenoma and correction of metabolic abnormalities in murine glycogen storage disease type Ia by gene therapy

1 reference

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9 September 2017

Long-term efficacy following readministration of an adeno-associated virus vector in dogs with glycogen storage disease type Ia

1 reference

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9 September 2017

Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector

1 reference

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9 September 2017

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

1 reference

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9 September 2017

A rapid and general assay for monitoring endogenous gene modification

1 reference

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9 September 2017

Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

9 September 2017

Complete normalization of hepatic G6PC deficiency in murine glycogen storage disease type Ia using gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

9 September 2017

Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

9 September 2017

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

9 September 2017

Emerging therapies for glycogen storage disease type I.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

9 September 2017

Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

9 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

9 September 2017

Sustained correction of bleeding disorder in hemophilia B mice by gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

9 September 2017

Glucose-6-phosphatase dependent substrate transport in the glycogen storage disease type-1a mouse

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

9 September 2017

Gene targeting to the ROSA26 locus directed by engineered zinc finger nucleases

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

29 September 2017

Gene delivery to the juvenile mouse liver using AAV2/8 vectors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

29 September 2017

Long-term correction of murine glycogen storage disease type Ia by recombinant adeno-associated virus-1-mediated gene transfer

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

29 September 2017

Targeted transgene insertion into the AAVS1 locus driven by baculoviral vector-mediated zinc finger nuclease expression in human-induced pluripotent stem cells.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

27 June 2018

Enhanced gene expression of systemically administered plasmid DNA in the liver with therapeutic ultrasound and microbubbles

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

4 September 2018

Comparison of gene transfer to the murine liver following intraperitoneal and intraportal delivery of hepatotropic AAV pseudo-serotypes.

1 reference

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4 September 2018

Activation of glycolysis and apoptosis in glycogen storage disease type Ia.

1 reference

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4 September 2018

AAV vector-mediated reversal of hypoglycemia in canine and murine glycogen storage disease type Ia.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

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Efficacy of helper-dependent adenovirus vector-mediated gene therapy in murine glycogen storage disease type Ia.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

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Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

4 September 2018

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1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4886939

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1 reference

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1 reference

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Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway

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1 reference

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12 December 2020

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10.1038/MT.2016.35

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7 March 2020

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Europe PubMed Central

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7 March 2020

PubMed publication ID

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Europe PubMed Central

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7 March 2020

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