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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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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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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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Low efficacy of recombinant SV40 in Ugt1a1-/- mice with severe inherited hyperbilirubinemia

PloS one, 2021-01, Vol.16 (4), p.e0250605-e0250605

2021

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A novel UGT1A1 gene mutation causing severe unconjugated hyperbilirubinemia: a case report

BMC pediatrics, 2019-05, Vol.19 (1), p.173-173, Article 173

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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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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Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients

Molecular therapy. Methods & clinical development, 2021-03, Vol.20, p.287-297

2021

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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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A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene

Molecular therapy. Methods & clinical development, 2020-09, Vol.18, p.250-258

2020

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A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

Molecular therapy. Methods & clinical development, 2016-01, Vol.3 (C), p.16049-16049, Article 16049

2016

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The wide spectrum of multidrug resistance 3 deficiency: From neonatal cholestasis to cirrhosis of adulthood

Gastroenterology (New York, N.Y. 1943), 2001-05, Vol.120 (6), p.1448-1458

2001

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Long-Term Effects of Biliverdin Reductase a Deficiency in Ugt1 -/- Mice: Impact on Redox Status and Metabolism

Antioxidants, 2021-12, Vol.10 (12), p.2029

2021

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What's next in gene therapy for Crigler-Najjar syndrome?

Expert opinion on biological therapy, 2023-02, Vol.23 (2), p.119-121

2023

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Therapeutic base editing in the adult liver

Nature reviews. Gastroenterology & hepatology, 2021-09, Vol.18 (9), p.597-598

2021

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The Genetic Basis of the Reduced Expression of Bilirubin UDP-Glucuronosyltransferase 1 in Gilbert's Syndrome

The New England journal of medicine, 1995-11, Vol.333 (18), p.1171-1175

1995

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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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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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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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Cre Recombinase Mediates the Removal of Bacterial Backbone to Efficiently Generate rSV40

Molecular therapy. Methods & clinical development, 2018-06, Vol.9, p.225-233

2018

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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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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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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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Ex-vivo evaluation of gene therapy vectors in human pancreatic (cancer) tissue slices

World journal of gastroenterology : WJG, 2009-03, Vol.15 (11), p.1359-1366

2009

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Disease burden and management of Crigler‐Najjar syndrome: Report of a world registry

Liver international, 2022-07, Vol.42 (7), p.1593-1604

2022

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

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

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

Low efficacy of recombinant SV40 in Ugt1a1-/- mice with severe inherited hyperbilirubinemia

A novel UGT1A1 gene mutation causing severe unconjugated hyperbilirubinemia: a case report

Biliverdin Reductase inhibitors did not improve severe unconjugated hyperbilirubinemia in vivo

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

Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients

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

A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

The wide spectrum of multidrug resistance 3 deficiency: From neonatal cholestasis to cirrhosis of adulthood

Long-Term Effects of Biliverdin Reductase a Deficiency in Ugt1 -/- Mice: Impact on Redox Status and Metabolism

What's next in gene therapy for Crigler-Najjar syndrome?

Therapeutic base editing in the adult liver

The Genetic Basis of the Reduced Expression of Bilirubin UDP-Glucuronosyltransferase 1 in Gilbert's Syndrome

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

ATP8B1 and ATP11C: Two Lipid Flippases Important for Hepatocyte Function

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

Cre Recombinase Mediates the Removal of Bacterial Backbone to Efficiently Generate rSV40

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

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

Anion Exchanger 2 Is Essential for Spermiogenesis in Mice

Ex-vivo evaluation of gene therapy vectors in human pancreatic (cancer) tissue slices

Disease burden and management of Crigler‐Najjar syndrome: Report of a world registry

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