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Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

Nature (London), 2014-02, Vol.506 (7488), p.382-386

2014

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Prevention of Cholestatic Liver Disease and Reduced Tumorigenicity in a Murine Model of PFIC Type 3 Using Hybrid AAV‐piggyBac Gene Therapy

Hepatology (Baltimore, Md.), 2019-12, Vol.70 (6), p.2047-2061

2019

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Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genome

Nature genetics, 2017-08, Vol.49 (8), p.1267-1273

2017

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ACE2 Therapy Using Adeno-associated Viral Vector Inhibits Liver Fibrosis in Mice

Molecular therapy, 2015-09, Vol.23 (9), p.1434-1443

2015

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Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors

Molecular therapy, 2008-06, Vol.16 (6), p.1081-1088

2008

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Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys

Molecular therapy. Methods & clinical development, 2021-12, Vol.23, p.135-146

2021

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Liver‐Targeted Angiotensin Converting Enzyme 2 Therapy Inhibits Chronic Biliary Fibrosis in Multiple Drug‐Resistant Gene 2‐Knockout Mice

Hepatology communications, 2019-12, Vol.3 (12), p.1656-1673

2019

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Modeling correction of severe urea cycle defects in the growing murine liver using a hybrid recombinant adeno‐associated virus/piggyBac transposase gene delivery system

Hepatology (Baltimore, Md.), 2015-08, Vol.62 (2), p.417-428

2015

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Adeno-associated Virus-mediated Rescue of Neonatal Lethality in Argininosuccinate Synthetase-deficient Mice

Molecular therapy, 2013-10, Vol.21 (10), p.1823-1831

2013

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Intrabiliary infusion of naked DNA vectors targets periportal hepatocytes in mice

Molecular therapy. Methods & clinical development, 2022-12, Vol.27, p.352-367

2022

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Induction and Prevention of Severe Hyperammonemia in the spfash Mouse Model of Ornithine Transcarbamylase Deficiency Using shRNA and rAAV-mediated Gene Delivery

Molecular therapy, 2011-05, Vol.19 (5), p.854-859

2011

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Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes

JHEP reports, 2020-02, Vol.2 (1), p.100065-100065, Article 100065

2020

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Use of a Hybrid Adeno-Associated Viral Vector Transposon System to Deliver the Insulin Gene to Diabetic NOD Mice

Cells (Basel, Switzerland), 2020-10, Vol.9 (10), p.2227

2020

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Insights into Gene Therapy for Urea Cycle Defects by Mathematical Modeling

Human gene therapy, 2019-11, Vol.30 (11), p.1385

2019

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Recapitulation of Skewed X-Inactivation in Female Ornithine Transcarbamylase-Deficient Primary Human Hepatocytes in the FRG Mouse: A Novel System for Developing Epigenetic Therapies

Human gene therapy, 2023-09, Vol.34 (17-18), p.917

2023

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AAV2/8-mediated Correction of OTC Deficiency Is Robust in Adult but Not Neonatal Spfash Mice

Molecular therapy, 2009-08, Vol.17 (8), p.1340-1346

2009

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AAV2/8-mediated correction of OTC deficiency is robust in adult but not neonatal Spf(ash) mice

Molecular therapy, 2009-08, Vol.17 (8), p.1340-1346

2009

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1778-P: Delivery of the Insulin Gene Using an Integrating Adeno-Associated Viral Vector (AAV) to Diabetic NOD Mice

Diabetes (New York, N.Y.), 2019-06, Vol.68 (Supplement_1)

2019

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Sexually Dimorphic Patterns of Episomal rAAV Genome Persistence in the Adult Mouse Liver and Correlation With Hepatocellular Proliferation

Molecular therapy, 2009-09, Vol.17 (9), p.1548-1554

2009

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Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders

Journal of inherited metabolic disease, 2012-07, Vol.35 (4), p.641-645

2012

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AAV vectors encoding malarial antigens stimulate antigen-specific immunity but do not protect from parasite infection

Vaccine, 2007-01, Vol.25 (6), p.1014-1022

2007

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Partial correction of sensitivity to oxidant stress in friedreich ataxia patient fibroblasts by frataxin-encoding adeno-associated virus and lentivirus vectors

Human gene therapy, 2005-08, Vol.16 (8), p.947-956

2005

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Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort

Human gene therapy, 2019-01, Vol.30 (1), p.79

2019

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Treatment of an infant with X‐linked severe combined immunodeficiency (SCID‐X1) by gene therapy in Australia

Medical journal of Australia, 2005-05, Vol.182 (9), p.458-463

2005

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Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

Prevention of Cholestatic Liver Disease and Reduced Tumorigenicity in a Murine Model of PFIC Type 3 Using Hybrid AAV‐piggyBac Gene Therapy

Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genome

ACE2 Therapy Using Adeno-associated Viral Vector Inhibits Liver Fibrosis in Mice

Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors

Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys

Liver‐Targeted Angiotensin Converting Enzyme 2 Therapy Inhibits Chronic Biliary Fibrosis in Multiple Drug‐Resistant Gene 2‐Knockout Mice

Modeling correction of severe urea cycle defects in the growing murine liver using a hybrid recombinant adeno‐associated virus/piggyBac transposase gene delivery system

Adeno-associated Virus-mediated Rescue of Neonatal Lethality in Argininosuccinate Synthetase-deficient Mice

Intrabiliary infusion of naked DNA vectors targets periportal hepatocytes in mice

Induction and Prevention of Severe Hyperammonemia in the spfash Mouse Model of Ornithine Transcarbamylase Deficiency Using shRNA and rAAV-mediated Gene Delivery

Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes

Use of a Hybrid Adeno-Associated Viral Vector Transposon System to Deliver the Insulin Gene to Diabetic NOD Mice

Insights into Gene Therapy for Urea Cycle Defects by Mathematical Modeling

Recapitulation of Skewed X-Inactivation in Female Ornithine Transcarbamylase-Deficient Primary Human Hepatocytes in the FRG Mouse: A Novel System for Developing Epigenetic Therapies

Sleeping Beauty mRNA-LNP enables stable rAAV transgene expression in mouse and NHP hepatocytes and improves vector potency

AAV2/8-mediated Correction of OTC Deficiency Is Robust in Adult but Not Neonatal Spfash Mice

AAV2/8-mediated correction of OTC deficiency is robust in adult but not neonatal Spf(ash) mice

1778-P: Delivery of the Insulin Gene Using an Integrating Adeno-Associated Viral Vector (AAV) to Diabetic NOD Mice

Sexually Dimorphic Patterns of Episomal rAAV Genome Persistence in the Adult Mouse Liver and Correlation With Hepatocellular Proliferation

Gene therapy for metabolic disorders: an overview with a focus on urea cycle disorders

AAV vectors encoding malarial antigens stimulate antigen-specific immunity but do not protect from parasite infection

Partial correction of sensitivity to oxidant stress in friedreich ataxia patient fibroblasts by frataxin-encoding adeno-associated virus and lentivirus vectors

Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort

Treatment of an infant with X‐linked severe combined immunodeficiency (SCID‐X1) by gene therapy in Australia

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