Hepatic lentiviral gene transfer prevents the long-term onset of hepatic tumours of glycogen storage disease type 1a in mice

Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy

(2014) N. Nair et al.   BLOOD

Liver-Specific Transcriptional Modules Identified by Genome-Wide In Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates

(2014) Marinee K Chuah et al.   MOLECULAR THERAPY

NAFLD leads to liver cancer: Do we have sufficient evidence?

(2013) Xiao-Yan Duan et al.   CANCER LETTERS

Biosafety Features of Lentiviral Vectors

(2013) Axel Schambach et al.   HUMAN GENE THERAPY

Monogenic diseases that can be cured by liver transplantation

(2013) Stefano Fagiuoli et al.   JOURNAL OF HEPATOLOGY

Hepatic Diseases Related to Triglyceride Metabolism

(2013) Asdrubal Aguilera-Méndez et al.   MINI-REVIEWS IN MEDICINAL CHEMISTRY

Prevention of hepatocellular adenoma and correction of metabolic abnormalities in murine glycogen storage disease type Ia by gene therapy

(2012) Young Mok Lee et al.   HEPATOLOGY

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

(2012) Ina Rittelmeyer et al.   HEPATOLOGY

Molecular characterization of hepatocellular adenomas developed in patients with glycogen storage disease type I

(2012) Julien Calderaro et al.   JOURNAL OF HEPATOLOGY

In search of proof-of-concept: gene therapy for glycogen storage disease type Ia

(2012) Dwight D. Koeberl   JOURNAL OF INHERITED METABOLIC DISEASE

Retroviral Vector-mediated Gene Therapy for Metabolic Diseases: An Update

(2011) Nicolas Ferry et al.   CURRENT PHARMACEUTICAL DESIGN

Recombinant AAV-directed gene therapy for type I glycogen storage diseases

(2011) Janice Y Chou et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Long-Term Efficacy Following Readministration of an Adeno-Associated Virus Vector in Dogs with Glycogen Storage Disease Type Ia

(2011) Amanda Demaster et al.   HUMAN GENE THERAPY

A MicroRNA-regulated and GP64-pseudotyped Lentiviral Vector Mediates Stable Expression of FVIII in a Murine Model of Hemophilia A

(2011) Hideto Matsui et al.   MOLECULAR THERAPY

Glucose-6-phosphatase deficiency

(2011) Roseline Froissart et al.   Orphanet Journal of Rare Diseases

Hepatocellular Carcinoma and Non-Alcoholic Fatty Liver Disease: From a Clinical to a Molecular Association

(2010) S. Petta et al.   CURRENT PHARMACEUTICAL DESIGN

Lentiviral Vectors That Express UGT1A1 in Liver and Contain miR-142 Target Sequences Normalize Hyperbilirubinemia in Gunn Rats

(2010) Françoise Schmitt et al.   GASTROENTEROLOGY

Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

(2010) Sylvie Boutin et al.   HUMAN GENE THERAPY

Targeted deletion of liver glucose-6 phosphatase mimics glycogen storage disease type 1a including development of multiple adenomas

(2010) Elodie Mutel et al.   JOURNAL OF HEPATOLOGY

Neonatal Gene Therapy of Glycogen Storage Disease Type Ia Using a Feline Immunodeficiency Virus–based Vector

(2010) Albert Grinshpun et al.   MOLECULAR THERAPY

Protein Feeding Promotes Redistribution of Endogenous Glucose Production to the Kidney and Potentiates Its Suppression by Insulin

(2008) Bruno Pillot et al.   ENDOCRINOLOGY

Potential of AAV vectors in the treatment of metabolic disease

(2008) I E Alexander et al.   GENE THERAPY