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

IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

(2020) Christian Leborgne et al.   NATURE MEDICINE

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

(2019) Fanny Collaud et al.   Molecular Therapy-Methods & Clinical Development

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

(2019) Sem J. Aronson et al.   HUMAN GENE THERAPY

Disease burden of Crigler–Najjar syndrome: systematic review and future perspectives

(2019) Anil Dhawan et al.   JOURNAL OF GASTROENTEROLOGY AND HEPATOLOGY

AAV8 Gene Therapy Rescues the Newborn Phenotype of a Mouse Model of Crigler–Najjar

(2018) Jenny A. Greig et al.   HUMAN GENE THERAPY

Age-related seroprevalence of antibodies against AAV-LK03 in a UK population cohort

(2018) Dany Perocheau et al.   HUMAN GENE THERAPY

Hepatic Parenchymal Injury in Crigler-Najjar Type I

(2018) Ellen Mitchell et al.   JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION

Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction

(2018) Zachary Fitzpatrick et al.   Molecular Therapy-Methods & Clinical Development

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

(2018) Pasqualina Colella et al.   Molecular Therapy-Methods & Clinical Development

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration

(2018) Amine Meliani et al.   Nature Communications

Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUgt1a1 enzyme in a mouse model of Crigler–Najjar Syndrome Type I

(2017) L Bočkor et al.   GENE THERAPY

Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors

(2017) Marianna Hösel et al.   HEPATOLOGY

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

(2017) Lindsey A. George et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2016) Giuseppe Ronzitti et al.   Molecular Therapy-Methods & Clinical Development

Synergy between rapamycin and FLT3 ligand enhances plasmacytoid dendritic cell-dependent induction of CD4+CD25+FoxP3+ Treg

(2015) M. Biswas et al.   BLOOD

Determination of Anti-Adeno-Associated Virus Vector Neutralizing Antibody Titer with anIn VitroReporter System

(2015) Amine Meliani et al.   Human Gene Therapy Methods

Liver Damage, Inflammation, and Enhanced Tumorigenesis after Persistent mTORC1 Inhibition

(2014) Atsushi Umemura et al.   Cell Metabolism

Inherited Disorders of Bilirubin Transport and Conjugation: New Insights Into Molecular Mechanisms and Consequences

(2014) Serge Erlinger et al.   GASTROENTEROLOGY

Life-Long Correction of Hyperbilirubinemia with a Neonatal Liver-Specific AAV-Mediated Gene Transfer in a Lethal Mouse Model of Crigler–Najjar Syndrome

(2014) Giulia Bortolussi et al.   HUMAN GENE THERAPY

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

In the rat liver, Adenoviral gene transfer efficiency is comparable to AAV

(2013) P S Montenegro-Miranda et al.   GENE THERAPY

Polyinosinic Acid Blocks Adeno-Associated Virus Macrophage Endocytosis In Vitro and Enhances Adeno-Associated Virus Liver-Directed Gene Therapy In Vivo

(2013) Remco van Dijk et al.   HUMAN GENE THERAPY

The prevalence of neutralizing antibodies against adeno-associated virus capsids is reduced in young Japanese individuals

(2013) Jun Mimuro et al.   JOURNAL OF MEDICAL VIROLOGY

Liver transplantation in Crigler-Najjar syndrome type I disease

(2012) Zhen-Hua Tu et al.   Hepatobiliary & Pancreatic Diseases International

Evolution of indications and results of liver transplantation in Europe. A report from the European Liver Transplant Registry (ELTR)

(2012) René Adam et al.   JOURNAL OF HEPATOLOGY

AAV vectors transduce hepatocytes in vivo as efficiently in cirrhotic as in healthy rat livers

(2011) L Sobrevals et al.   GENE THERAPY

A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8

(2011) Virginie Monteilhet et al.   MOLECULAR THERAPY

Gender dependence in the hormone content of the immune cells

(2009) Gy Csaba et al.   ACTA PHYSIOLOGICA HUNGARICA

Macrophages

(2009) Barbro N. Melgert et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Effect of Adeno-Associated Virus Serotype and Genomic Structure on Liver Transduction and Biodistribution in Mice of Both Genders

(2009) Astrid Pañeda et al.   HUMAN GENE THERAPY

Transient expression of genes delivered to newborn rat liver using recombinant adeno-associated virus 2/8 vectors

(2009) Maude Flageul et al.   JOURNAL OF GENE MEDICINE

mTOR Regulation and Therapeutic Rejuvenation of Aging Hematopoietic Stem Cells

(2009) C. Chen et al.   Science Signaling

Complement Is an Essential Component of the Immune Response to Adeno-Associated Virus Vectors

(2008) A. K. Zaiss et al.   JOURNAL OF VIROLOGY