Liver gene therapy: advances and hurdles (Q35904443)

11 August 2017

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title

Liver gene therapy: advances and hurdles (English)

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11 August 2017

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Nguyen TH

1

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PubMed ID

15454961

11 August 2017

Ferry N

series ordinal

2

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11 August 2017

1 October 2004

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11 August 2017

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11 August 2017

11 Suppl 1

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11 August 2017

S76-84

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https://scigraph.springernature.com/pub.10.1038/sj.gt.3302373

11 August 2017

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cites work

Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinaemia type I.

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https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Correction of liver disease by hepatocyte transplantation in a mouse model of progressive familial intrahepatic cholestasis.

21 January 2018

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Adult-to-adult living-donor liver transplantation. A summary of current status and an outline of the program in Geneva

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Potential impact of in situ liver splitting on the number of available grafts

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

The basic science of gene therapy

21 January 2018

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https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Fusion of bone-marrow-derived cells with Purkinje neurons, cardiomyocytes and hepatocytes

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Transplanted bone marrow regenerates liver by cell fusion

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Cell fusion is the principal source of bone-marrow-derived hepatocytes

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Overview of recent experimental studies on liver stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

In vitro and in vivo hepatoma cell-specific expression of a gene transferred with an adenoviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

A blood-tumor barrier limits gene transfer to experimental liver cancer: the effect of vasoactive compounds.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Enhanced in vivo adenovirus-mediated gene transfer to rat hepatocarcinomas by selective administration into the hepatic artery

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Microtubular disruption prolongs the expression of human bilirubin-uridinediphosphoglucuronate-glucuronosyltransferase-1 gene transferred into Gunn rat livers

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Gene transfer by DNA/glycosylated polylysine complexes into human blood monocyte-derived macrophages

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Progress and prospects: naked DNA gene transfer and therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Adenovirus-mediated in vivo gene transfer and expression in normal rat liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Assessment of recombinant adenoviral vectors for hepatic gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Cyclophosphamide diminishes inflammation and prolongs transgene expression following delivery of adenoviral vectors to mouse liver and lung.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Transient immunomodulation with anti-CD40 ligand antibody and CTLA4Ig enhances persistence and secondary adenovirus-mediated gene transfer into mouse liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Transient immunosuppression with FK506 permits long-term expression of therapeutic genes introduced into the liver using recombinant adenoviruses in the rat.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Insertion of the adenoviral E3 region into a recombinant viral vector prevents antiviral humoral and cellular immune responses and permits long-term gene expression

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Helper-dependent adenoviral vectors mediate therapeutic factor VIII expression for several months with minimal accompanying toxicity in a canine model of severe hemophilia A

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

CMV-beta-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1 alpha promoter and results in therapeutic levels of human factor X in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Successful interference with cellular immune responses to immunogenic proteins encoded by recombinant viral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Highly efficient lentiviral vector-mediated transduction of nondividing, fully reimplantable primary hepatocytes.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Role of hepatocyte direct hyperplasia in lentivirus-mediated liver transduction in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Efficient lentiviral transduction of liver requires cell cycling in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Retroviral-mediated gene transfer into hepatocytes in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

In vivo retrovirus-mediated gene transfer into lamb liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Highly efficient retrovirus-mediated gene transfer into rat hepatocytes in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Proliferation induced by keratinocyte growth factor enhances in vivo retroviral-mediated gene transfer to mouse hepatocytes.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Portal branch occlusion safely facilitates in vivo retroviral vector transduction of rat liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Efficient retroviral gene transfer to the liver in vivo using nonpolypeptidic mitogens

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Factors influencing immune response after in vivo retrovirus-mediated gene transfer to the liver.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Cytotoxic Immune Response After Retroviral-Mediated Hepatic Gene Transfer in Rat Does Not Preclude Expression from Adeno-Associated Virus 1 Transduced Muscles

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Long-Term Improvement of Hypercholesterolemia After Ex Vivo Gene Therapy in LDLR-Deficient Rabbits

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Gene therapy for cholesterol

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Complete reconstitution of mouse liver with xenogeneic hepatocytes

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

A highly efficient, stable, and rapid approach for ex vivo human liver gene therapy via a FLAP lentiviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Treatment of the Crigler-Najjar syndrome type I with hepatocyte transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Isolated hepatocyte transplantation in an infant with a severe urea cycle disorder.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Therapeutic potential of hepatocyte transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Hepatocyte transplantation in a 4-year-old girl with peroxisomal biogenesis disease: technique, safety, and metabolic follow-up

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Hepatocyte transplantation as a treatment for glycogen storage disease type 1a.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Permanent engraftment and function of hepatocytes delivered to the liver: implications for gene therapy and liver repopulation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Mouse hepatocytes migrate to liver parenchyma and function indefinitely after intrasplenic transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Studies of liver repopulation using the dipeptidyl peptidase IV-deficient rat and other rodent recipients: cell size and structure relationships regulate capacity for increased transplanted hepatocyte mass in the liver lobule

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Kupffer cells participate in early clearance of syngeneic hepatocytes transplanted in the rat liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Treatment of hypercholesterolemia in the Watanabe rabbit using allogeneic hepatocellular transplantation under a regeneration stimulus

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Long-term, near-total liver replacement by transplantation of isolated hepatocytes in rats treated with retrorsine

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Cyclophosphamide disrupts hepatic sinusoidal endothelium and improves transplanted cell engraftment in rat liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Hepatic sinusoidal vasodilators improve transplanted cell engraftment and ameliorate microcirculatory perturbations in the liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Liver repopulation after cell transplantation in mice treated with retrorsine and carbon tetrachloride

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

A novel strategy for in vivo expansion of transplanted hepatocytes using preparative hepatic irradiation and FasL-induced hepatocellular apoptosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Cell transplantation after oxidative hepatic preconditioning with radiation and ischemia-reperfusion leads to extensive liver repopulation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Repopulation of rat liver by fetal hepatoblasts and adult hepatocytes transduced ex vivo with lentiviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Selective repopulation of normal mouse liver by Fas/CD95-resistant hepatocytes

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Loss of p27(Kip1) enhances the transplantation efficiency of hepatocytes transferred into diseased livers

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Normal hepatocytes correct serum bilirubin after repopulation of Gunn rat liver subjected to irradiation/partial resection

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Correction of liver disease following transplantation of normal rat hepatocytes into Long-Evans Cinnamon rats modeling Wilson's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

Intrahepatic transplantation of normal hepatocytes prevents Wilson's disease in Long-Evans cinnamon rats

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

RNA interference targeting Fas protects mice from fulminant hepatitis

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

Cytotoxic T-lymphocyte target proteins and their major histocompatibility complex class I restriction in response to adenovirus vectors delivered to mouse liver.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

AAV-mediated gene transfer for hemophilia

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

In vivo retrovirus-mediated gene transfer to the liver of dogs results in transient expression and induction of a cytotoxic immune response

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

A model for directed foreign gene delivery to rat liver cells in vivo

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

Cytotoxic immune response blunts long-term transgene expression after efficient retroviral-mediated hepatic gene transfer in rat

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

Cardiovascular features of homozygous familial hypercholesterolemia: Analysis of 16 patients

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

Entry and integration of transplanted hepatocytes in rat liver plates occur by disruption of hepatic sinusoidal endothelium

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

Transplanted reporter cells help in defining onset of hepatocyte proliferation during the life of F344 rats

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

Efficacy and safety of repeated hepatocyte transplantation for significant liver repopulation in rodents

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

Sinusoidal ultrastructure evaluated during the revascularization of regenerating rat liver

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

Hepatocyte transplantation in a model of toxin-induced liver disease: variable therapeutic effect during replacement of damaged parenchyma by donor cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

Role of thyroid hormone in stimulating liver repopulation in the rat by transplanted hepatocytes

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

Selective repopulation of mice liver after Fas-resistant hepatocyte transplantation

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

Therapeutic liver repopulation in a mouse model of hypercholesterolemia

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302373

7 January 2021

Identifiers

DOI

10.1038/SJ.GT.3302373

1 reference

11 August 2017

1 reference

PubMed ID

15454961