(Q30856034)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27464750%20AND%20SRC:MED&resulttype=core&format=json

19 March 2020

title

Curative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1 (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27464750%20AND%20SRC:MED&resulttype=core&format=json

19 March 2020

0 references

9

1 reference

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19 March 2020

Markus Grompe

18

1 reference

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19 March 2020

21

Stephen J Russell

1 reference

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19 March 2020

15

Kah Whye Peng

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27464750%20AND%20SRC:MED&resulttype=core&format=json

19 March 2020

11

Lukkana Suksanpaisan

1 reference

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19 March 2020

2

Shennen A Mao

1 reference

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19 March 2020

3

Jaime Glorioso

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27464750%20AND%20SRC:MED&resulttype=core&format=json

19 March 2020

5

Bruce Amiot

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27464750%20AND%20SRC:MED&resulttype=core&format=json

19 March 2020

10

Timothy R DeGrado

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27464750%20AND%20SRC:MED&resulttype=core&format=json

19 March 2020

22

Scott L Nyberg

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27464750%20AND%20SRC:MED&resulttype=core&format=json

19 March 2020

author name string

Raymond D Hickey

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27464750%20AND%20SRC:MED&resulttype=core&format=json

19 March 2020

Faysal Elgilani

4

1 reference

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19 March 2020

Harvey Chen

6

1 reference

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19 March 2020

Piero Rinaldo

7

1 reference

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19 March 2020

Ronald Marler

8

1 reference

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19 March 2020

Michael K O'Connor

12

1 reference

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19 March 2020

Brittany L Freeman

13

1 reference

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19 March 2020

Samar H Ibrahim

14

1 reference

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19 March 2020

Cary O Harding

16

1 reference

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19 March 2020

Chak-Sum Ho

17

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27464750%20AND%20SRC:MED&resulttype=core&format=json

19 March 2020

Yasuhiro Ikeda

19

1 reference

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19 March 2020

Joseph B Lillegard

20

1 reference

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19 March 2020

language of work or name

English

0 references

publication date

1 July 2016

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27464750%20AND%20SRC:MED&resulttype=core&format=json

Science Translational Medicine

19 March 2020

published in

1 reference

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19 March 2020

volume

8

1 reference

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19 March 2020

issue

349

1 reference

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19 March 2020

page(s)

349ra99

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27464750%20AND%20SRC:MED&resulttype=core&format=json

Liver-directed lentiviral gene therapy in a dog model of hemophilia B.

19 March 2020

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy.

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Noninvasive 3-dimensional imaging of liver regeneration in a mouse model of hereditary tyrosinemia type 1 using the sodium iodide symporter gene

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Cross-sectional study of 168 patients with hepatorenal tyrosinaemia and implications for clinical practice

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Fumarylacetoacetate hydrolase deficient pigs are a novel large animal model of metabolic liver disease

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Targeted disruption of LDLR causes hypercholesterolemia and atherosclerosis in Yucatan miniature pigs

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Mixed lineage kinase 3 deficient mice are protected against the high fat high carbohydrate diet-induced steatohepatitis.

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Hepatocyte transplantation for inherited metabolic diseases of the liver.

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Lentiviral vectors: basic to translational

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Rescue of lethal hepatic failure by hepatized lymph nodes in mice

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Synthesis and biological evaluation of [(18)F]tetrafluoroborate: a PET imaging agent for thyroid disease and reporter gene imaging of the sodium/iodide symporter

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Cellular loss after allogenic hepatocyte transplantation

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Hepatic irradiation augments engraftment of donor cells following hepatocyte transplantation

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Cell-mediated rejection results in allograft loss after liver cell transplantation

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Homogentisate metabolism: the isomerization of maleylacetoacetate by an enzyme which requires glutathione

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Kinetics of liver repopulation after bone marrow transplantation.

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Tyrosinaemia type I and apoptosis of hepatocytes and renal tubular cells

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Hepatocyte injury in tyrosinemia type 1 is induced by fumarylacetoacetate and is inhibited by caspase inhibitors

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

The mutagenicity of the tyrosine metabolite, fumarylacetoacetate, is enhanced by glutathione depletion

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Hereditary tyrosinemia type I. Self-induced correction of the fumarylacetoacetase defect

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Loss of fumarylacetoacetate hydrolase is responsible for the neonatal hepatic dysfunction phenotype of lethal albino mice.

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Visceral pathology of hereditary tyrosinemia type I.

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Long-Term Improvement of Hypercholesterolemia After Ex Vivo Gene Therapy in LDLR-Deficient Rabbits

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Treatment of hereditary tyrosinaemia type I by inhibition of 4-hydroxyphenylpyruvate dioxygenase

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Correction of hyperbilirubinemia in the glucuronyltransferase-deficient rat by intraportal hepatocyte transplantation

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

On the enzymic defects in hereditary tyrosinemia

28 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Efficient production of Fah-null heterozygote pigs by chimeric adeno-associated virus-mediated gene knockout and somatic cell nuclear transfer.

28 July 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAF3838

Hepatic lentiviral gene transfer is associated with clonal selection, but not with tumor formation in serially transplanted rodents.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAF3838

Molecular characterization of swine leucocyte antigen class I genes in outbred pig populations.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAF3838

Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAF3838

Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinaemia type I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAF3838

Ex vivo hepatic gene therapy of a mouse model of Hereditary Tyrosinemia Type I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAF3838

Metabolic liver disease in children.

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAF3838

Single liver lobe repopulation with wildtype hepatocytes using regional hepatic irradiation cures jaundice in Gunn rats

21 January 2018

1 reference

https://api.crossref.org/works/10.1126%2FSCITRANSLMED.AAF3838

A technique for porcine hepatocyte harvest and description of differentiated metabolic functions in static culture.

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5477771

Molecular characterization of swine leucocyte antigen class II genes in outbred pig populations

25 November 2018

1 reference

12 December 2020

Regional transient portal ischemia and irradiation as preparative regimen for hepatocyte transplantation

1 reference

12 December 2020

Current status of hepatocyte transplantation

1 reference

12 December 2020

Hepatocellular transplantation for metabolic deficiencies: decrease of plasms bilirubin in Gunn rats

1 reference

12 December 2020

10.1126/SCITRANSLMED.AAF3838

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27464750%20AND%20SRC:MED&resulttype=core&format=json

19 March 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27464750%20AND%20SRC:MED&resulttype=core&format=json

19 March 2020

1 reference