Synthetic human ABCB4 mRNA therapy rescues severe liver disease phenotype in a BALB/c.Abcb4 mouse model of PFIC3

Durable anticancer immunity from intratumoral administration of IL-23, IL-36γ, and OX40L mRNAs

(2019) Susannah L. Hewitt et al.   Science Translational Medicine

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

(2019) Sem J. Aronson et al.   JOURNAL OF HEPATOLOGY

Prevention of cholestatic liver disease and reduced tumorigenicity in a murine model of PFIC type 3 using hybrid AAV‐ piggyBac gene therapy

(2019) S.M Siew et al.   HEPATOLOGY

mRNA Therapy Improves Metabolic and Behavioral Abnormalities in a Murine Model of Citrin Deficiency

(2019) Jingsong Cao et al.   MOLECULAR THERAPY

Clinical utility of genomic analysis in adults with idiopathic liver disease

(2019) Aaron Hakim et al.   JOURNAL OF HEPATOLOGY

Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model

(2019) Nicholas D. Weber et al.   Nature Communications

A Novel Amino Lipid Series for mRNA Delivery: Improved Endosomal Escape and Sustained Pharmacology and Safety in Non-human Primates

(2018) Staci Sabnis et al.   MOLECULAR THERAPY

Systemic messenger RNA as an etiological treatment for acute intermittent porphyria

(2018) Lei Jiang et al.   NATURE MEDICINE

ABCB4 Gene Aberrations in Human Liver Disease: An Evolving Spectrum

(2018) Matthias Reichert et al.   SEMINARS IN LIVER DISEASE

The ABCB4 p.T175A variant as potential modulator of hepatic fibrosis in patients with chronic liver diseases: Looking beyond the cholestatic realm

(2017) Marcin Krawczyk et al.   HEPATOLOGY

Biliary bile acids in hepatobiliary injury – What is the link?

(2017) Peter Fickert et al.   JOURNAL OF HEPATOLOGY

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

(2017) Ding An et al.   Cell Reports

A New Mdr2−/− Mouse Model of Sclerosing Cholangitis with Rapid Fibrosis Progression, Early-Onset Portal Hypertension, and Liver Cancer

(2015) Naoki Ikenaga et al.   AMERICAN JOURNAL OF PATHOLOGY

Lysosomal Storage Diseases: From Pathophysiology to Therapy

(2015) Giancarlo Parenti et al.   Annual Review of Medicine

A functional classification ofABCB4variations causing progressive familial intrahepatic cholestasis type 3

(2015) Jean-Louis Delaunay et al.   HEPATOLOGY

Integrin αvβ6 critically regulates hepatic progenitor cell function and promotes ductular reaction, fibrosis, and tumorigenesis

(2015) Zhen-Wei Peng et al.   HEPATOLOGY

Large-scale whole-genome sequencing of the Icelandic population

(2015) Daniel F Gudbjartsson et al.   NATURE GENETICS

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

mRNA-based therapeutics — developing a new class of drugs

(2014) Ugur Sahin et al.   NATURE REVIEWS DRUG DISCOVERY

A critical analysis of codon optimization in human therapeutics

(2014) Vincent P. Mauro et al.   TRENDS IN MOLECULAR MEDICINE

Failure of Fibrotic Liver Regeneration in Mice Is Linked to a Severe Fibrogenic Response Driven by Hepatic Progenitor Cell Activation

(2013) Kaori Kuramitsu et al.   AMERICAN JOURNAL OF PATHOLOGY

Fate tracing reveals hepatic stellate cells as dominant contributors to liver fibrosis independent of its aetiology

(2013) Ingmar Mederacke et al.   Nature Communications

Progressive Familial Intrahepatic Cholestasis (PFIC) Type 1, 2, and 3: A Review of the Liver Pathology Findings

(2011) Raffaella Morotti et al.   SEMINARS IN LIVER DISEASE

Macrophage-mediated phagocytosis of apoptotic cholangiocytes contributes to reversal of experimental biliary fibrosis

(2010) Yury Popov et al.   AMERICAN JOURNAL OF PHYSIOLOGY-GASTROINTESTINAL AND LIVER PHYSIOLOGY

Progressive familial intrahepatic cholestasis: a single-center experience of living-donor liver transplantation during two decades in Japan

(2010) Tomohide Hori et al.   CLINICAL TRANSPLANTATION

Clinical Features and Genotype-Phenotype Correlations in Children With Progressive Familial Intrahepatic Cholestasis Type 3 Related to ABCB4 Mutations

(2010) Carla Colombo et al.   JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION

The Spectrum of Liver Diseases Related toABCB4Gene Mutations: Pathophysiology and Clinical Aspects

(2010) Anne Davit-Spraul et al.   SEMINARS IN LIVER DISEASE

Liver diseases related to MDR3 (ABCB4) gene deficiency

(2009) Emmanuel Gonzales   Frontiers in Bioscience-Landmark

Hepatic venous pressure gradient predicts development of hepatocellular carcinoma independently of severity of cirrhosis

(2009) Cristina Ripoll et al.   JOURNAL OF HEPATOLOGY

ABCB4 Heterozygous Gene Mutations Associated With Fibrosing Cholestatic Liver Disease in Adults

(2008) Marianne Ziol et al.   GASTROENTEROLOGY

Integrin αvβ6 is a marker of the progression of biliary and portal liver fibrosis and a novel target for antifibrotic therapies

(2008) Yury Popov et al.   JOURNAL OF HEPATOLOGY