Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A

The aftermath of the interplay between the endoplasmic reticulum stress response and redox signaling

(2021) Kashi Raj Bhattarai et al.   EXPERIMENTAL AND MOLECULAR MEDICINE

Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A

(2021) K John Pasi et al.   HAEMOPHILIA

Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers

(2021) Choong-Ryoul Sihn et al.   Molecular Therapy-Methods & Clinical Development

Factor VIII exhibits chaperone-dependent and glucose-regulated reversible amyloid formation in the endoplasmic reticulum

(2020) Juthakorn Poothong et al.   BLOOD

Role of Essential Metal Ions in AAV Vector-Mediated Transduction

(2020) Himanshu K. Rambhai et al.   Molecular Therapy-Methods & Clinical Development

WFH Guidelines for the Management of Hemophilia, 3rd edition

(2020) Alok Srivastava et al.   HAEMOPHILIA

Activity of Transgene-Produced B-Domain Deleted Factor VIII in Human Plasma Following AAV5 Gene Therapy

(2020) Steffen Rosen et al.   BLOOD

Induction of ER Stress by an AAV5 BDD FVIII Construct Is Dependent on the Strength of the Hepatic-Specific Promoter

(2020) Sylvia Fong et al.   Molecular Therapy-Methods & Clinical Development

A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells

(2020) Giang N. Nguyen et al.   NATURE BIOTECHNOLOGY

Adeno-associated virus vector as a platform for gene therapy delivery

(2019) Dan Wang et al.   NATURE REVIEWS DRUG DISCOVERY

The endothelial lectin clearance receptor CLEC4M binds and internalizes factor VIII in a VWF-dependent and independent manner

(2019) Laura L. Swystun et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Regulation of molecular chaperone GRP78 by HBV: control of viral replication and cell survival

(2019) Wangqin Shu et al.   MOLECULAR AND CELLULAR BIOLOGY

Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice

(2018) Stuart Bunting et al.   MOLECULAR THERAPY

AAV delivery of GRP78/BiP promotes adaptation of human RPE cell to ER stress

(2017) Shima Ghaderi et al.   JOURNAL OF CELLULAR BIOCHEMISTRY

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

(2017) Savita Rangarajan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Recombinant Adeno-Associated Virus Vector Genomes Take the Form of Long-Lived, Transcriptionally Competent Episomes in Human Muscle

(2016) Bruce C. Schnepp et al.   HUMAN GENE THERAPY

Adeno-Associated Virus Gene Therapy for Liver Disease

(2016) Lisa M. Kattenhorn et al.   HUMAN GENE THERAPY

Recombinant AAV Integration Is Not Associated With Hepatic Genotoxicity in Nonhuman Primates and Patients

(2016) Irene Gil-Farina et al.   MOLECULAR THERAPY

Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice

(2016) Amy M Lange et al.   Molecular Therapy-Methods & Clinical Development

Potential for cellular stress response to hepatic factor VIII expression from AAV vector

(2016) Irene Zolotukhin et al.   Molecular Therapy-Methods & Clinical Development

FcRn Rescues Recombinant Factor VIII Fc Fusion Protein from a VWF Independent FVIII Clearance Pathway in Mouse Hepatocytes

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Prevalence of Nonalcoholic Fatty Liver Disease in the United States: The Third National Health and Nutrition Examination Survey, 1988–1994

(2013) Mariana Lazo et al.   AMERICAN JOURNAL OF EPIDEMIOLOGY

Oversized AAV Transductifon Is Mediated via a DNA-PKcs-independent, Rad51C-dependent Repair Pathway

(2013) Matthew L Hirsch et al.   MOLECULAR THERAPY

Presence and severity of non-alcoholic fatty liver disease in a large prospective primary care cohort

(2011) Matthew J. Armstrong et al.   JOURNAL OF HEPATOLOGY

Prevalence of Nonalcoholic Fatty Liver Disease and Nonalcoholic Steatohepatitis Among a Largely Middle-Aged Population Utilizing Ultrasound and Liver Biopsy: A Prospective Study

(2010) Christopher D. Williams et al.   GASTROENTEROLOGY

Histologic Abnormalities are Common in Protocol Liver Allograft Biopsies From Patients With Normal Liver Function Tests

(2009) Susan C. Abraham et al.   AMERICAN JOURNAL OF SURGICAL PATHOLOGY

Genome-wide Mapping of HATs and HDACs Reveals Distinct Functions in Active and Inactive Genes

(2009) Zhibin Wang et al.   CELL

Systematic and integrative analysis of large gene lists using DAVID bioinformatics resources

(2009) Da Wei Huang et al.   Nature Protocols

Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle

(2008) M. Penaud-Budloo et al.   JOURNAL OF VIROLOGY

Bioinformatics enrichment tools: paths toward the comprehensive functional analysis of large gene lists

(2008) Da Wei Huang et al.   NUCLEIC ACIDS RESEARCH

Antioxidants reduce endoplasmic reticulum stress and improve protein secretion

(2008) J. D. Malhotra et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA