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Liver-directed gene therapy results in long-term correction of progressive familial intrahepatic cholestasis type 3 in mice

Journal of hepatology, 2019-07, Vol.71 (1), p.153-162

2019

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Gene Therapy for Progressive Familial Intrahepatic Cholestasis: Current Progress and Future Prospects

International journal of molecular sciences, 2020-12, Vol.22 (1), p.273

2020

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Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome

Human gene therapy, 2019-10, Vol.30 (10), p.1297-1305

2019

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Identification and quantification of defective virus genomes in high throughput sequencing data using DVG-profiler, a novel post-sequence alignment processing algorithm

PloS one, 2019-05, Vol.14 (5), p.e0216944-e0216944

2019

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The P4-ATPase ATP9A is a novel determinant of exosome release

PloS one, 2019-04, Vol.14 (4), p.e0213069-e0213069

2019

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Ins1-Cre and Ins1-CreER Gene Replacement Alleles Are Susceptible To Silencing By DNA Hypermethylation

Endocrinology (Philadelphia), 2020-08, Vol.161 (8)

2020

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Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome

Molecular therapy. Methods & clinical development, 2019-03, Vol.12, p.157-174

2019

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Biliverdin Reductase inhibitors did not improve severe unconjugated hyperbilirubinemia in vivo

Scientific reports, 2017-05, Vol.7 (1), p.1646-9, Article 1646

2017

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Pancreatic islet beta cell-specific deletion of G6pc2 reduces fasting blood glucose

Journal of molecular endocrinology, 2020-05, Vol.64 (4), p.235-248

2020

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ATP11C targets basolateral bile salt transporter proteins in mouse central hepatocytes

Hepatology (Baltimore, Md.), 2016-07, Vol.64 (1), p.161-174

2016

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Mutations in the MDR3 Gene Cause Progressive Familial Intrahepatic Cholestasis

Proceedings of the National Academy of Sciences - PNAS, 1998-01, Vol.95 (1), p.282-287

1998

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Adeno-associated viral vector serotype 5 poorly transduces liver in rat models

PloS one, 2013-12, Vol.8 (12), p.e82597-e82597

2013

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Correction of liver disease by hepatocyte transplantation in a mouse model of progressive familial intrahepatic cholestasis

Gastroenterology (New York, N.Y. 1943), 2000-12, Vol.119 (6), p.1720-1730

2000

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Congenital Jaundice in Rats with a Mutation in a Multidrug Resistance- Associated Protein Gene

Science (American Association for the Advancement of Science), 1996-02, Vol.271 (5252), p.1126-1128

1996

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Zonal down‐regulation and redistribution of the multidrug resistance protein 2 during bile duct ligation in rat liver

Hepatology (Baltimore, Md.), 2000-03, Vol.31 (3), p.684-693

2000

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Anion Exchanger 2 Is Essential for Spermiogenesis in Mice

Proceedings of the National Academy of Sciences - PNAS, 2003-12, Vol.100 (26), p.15847-15852

2003

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catalytic subunit of DNA-protein kinase (DNA-PKcs) is not required for Ig class-switch recombination

Proceedings of the National Academy of Sciences - PNAS, 2007-02, Vol.104 (8), p.2843-2848

2007

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Bilirubin UDP-glucuronosyltransferase 1 is the only relevant bilirubin glucuronidating isoform in man

The Journal of biological chemistry, 1994-07, Vol.269 (27), p.17960-17964

1994

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ATP8B1 and ATP11C: Two Lipid Flippases Important for Hepatocyte Function

Digestive diseases (Basel), 2015-01, Vol.33 (3), p.314-318

2015

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The Scid Mouse Mutant: Definition, Characterization, and Potential Uses

Annual review of immunology, 1991-01, Vol.9 (1), p.323-350

1991

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Nonsynonymous single-nucleotide polymorphisms in the G6PC2 gene affect protein expression, enzyme activity, and fasting blood glucose

The Journal of biological chemistry, 2022-02, Vol.298 (2), p.101534-101534, Article 101534

2022

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DNA-dependent protein kinase activity is not required for immunoglobulin class switching

The Journal of experimental medicine, 2002-12, Vol.196 (11), p.1483-1495

2002

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Ephrin A2 receptor targeting does not increase adenoviral pancreatic cancer transduction in vivo

World journal of gastroenterology : WJG, 2009-06, Vol.15 (22), p.2754-2762

2009

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Overexpression of insulin like growth factor binding protein 5 reduces liver fibrosis in chronic cholangiopathy

Biochimica et biophysica acta, 2012-06, Vol.1822 (6), p.996-1003

2012

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Glucose-6-phosphatase catalytic subunit 2 negatively regulates glucose oxidation and insulin secretion in pancreatic β-cells

The Journal of biological chemistry, 2022-04, Vol.298 (4), p.101729-101729, Article 101729

2022

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Liver-directed gene therapy results in long-term correction of progressive familial intrahepatic cholestasis type 3 in mice

Gene Therapy for Progressive Familial Intrahepatic Cholestasis: Current Progress and Future Prospects

Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome

Identification and quantification of defective virus genomes in high throughput sequencing data using DVG-profiler, a novel post-sequence alignment processing algorithm

The P4-ATPase ATP9A is a novel determinant of exosome release

Ins1-Cre and Ins1-CreER Gene Replacement Alleles Are Susceptible To Silencing By DNA Hypermethylation

Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome

Biliverdin Reductase inhibitors did not improve severe unconjugated hyperbilirubinemia in vivo

Pancreatic islet beta cell-specific deletion of G6pc2 reduces fasting blood glucose

ATP11C targets basolateral bile salt transporter proteins in mouse central hepatocytes

Mutations in the MDR3 Gene Cause Progressive Familial Intrahepatic Cholestasis

Adeno-associated viral vector serotype 5 poorly transduces liver in rat models

Correction of liver disease by hepatocyte transplantation in a mouse model of progressive familial intrahepatic cholestasis

Congenital Jaundice in Rats with a Mutation in a Multidrug Resistance- Associated Protein Gene

Zonal down‐regulation and redistribution of the multidrug resistance protein 2 during bile duct ligation in rat liver

Anion Exchanger 2 Is Essential for Spermiogenesis in Mice

catalytic subunit of DNA-protein kinase (DNA-PKcs) is not required for Ig class-switch recombination

Bilirubin UDP-glucuronosyltransferase 1 is the only relevant bilirubin glucuronidating isoform in man

ATP8B1 and ATP11C: Two Lipid Flippases Important for Hepatocyte Function

The Scid Mouse Mutant: Definition, Characterization, and Potential Uses

Nonsynonymous single-nucleotide polymorphisms in the G6PC2 gene affect protein expression, enzyme activity, and fasting blood glucose

DNA-dependent protein kinase activity is not required for immunoglobulin class switching

Ephrin A2 receptor targeting does not increase adenoviral pancreatic cancer transduction in vivo

Overexpression of insulin like growth factor binding protein 5 reduces liver fibrosis in chronic cholangiopathy

Glucose-6-phosphatase catalytic subunit 2 negatively regulates glucose oxidation and insulin secretion in pancreatic β-cells

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