(Q40342200)

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Successful treatment of UGT1A1 deficiency in a rat model of Crigler-Najjar disease by intravenous administration of a liver-specific lentiviral vector

scientific article published on 5 December 2005

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scholarly article

1 reference

Europe PubMed Central

PubMed publication ID

19 September 2017

Successful treatment of UGT1A1 deficiency in a rat model of Crigler-Najjar disease by intravenous administration of a liver-specific lentiviral vector (English)

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Europe PubMed Central

PubMed publication ID

19 September 2017

vector-borne disease

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author name string

Pascal van der Wegen

1

1 reference

Europe PubMed Central

PubMed publication ID

19 September 2017

Rogier Louwen

2

1 reference

Europe PubMed Central

PubMed publication ID

19 September 2017

Ali M Imam

3

1 reference

Europe PubMed Central

PubMed publication ID

19 September 2017

Ruvalic M Buijs-Offerman

4

1 reference

Europe PubMed Central

PubMed publication ID

19 September 2017

Maarten Sinaasappel

5

1 reference

Europe PubMed Central

PubMed publication ID

19 September 2017

Frank Grosveld

6

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Europe PubMed Central

PubMed publication ID

19 September 2017

Bob J Scholte

7

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Europe PubMed Central

PubMed publication ID

19 September 2017

publication date

5 December 2005

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19 September 2017

Molecular Therapy

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19 September 2017

13

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Europe PubMed Central

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19 September 2017

2

1 reference

Europe PubMed Central

PubMed publication ID

19 September 2017

374-381

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Europe PubMed Central

PubMed publication ID

19 September 2017

Treatment of Crigler-Najjar type 1 disease: relevance of early liver transplantation

1 reference

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Long term correction of bilirubin-UDP-glucuronosyltransferase deficiency in Gunn rats by administration of a recombinant adenovirus during the neonatal period

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Induction of central tolerance by intrathymic inoculation of adenoviral antigens into the host thymus permits long-term gene therapy in Gunn rats

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7 January 2021

Gene therapy with bilirubin-UDP-glucuronosyltransferase in the Gunn rat model of Crigler-Najjar syndrome type 1.

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7 January 2021

A non-immunogenic adenoviral vector, coexpressing CTLA4Ig and bilirubin-uridine-diphosphoglucuronateglucuronosyltransferase permits long-term, repeatable transgene expression in the Gunn rat model of Crigler-Najjar syndrome

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7 January 2021

Long-term reduction of serum bilirubin levels in Gunn rats by retroviral gene transfer in vivo

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Acute cytokine response to systemic adenoviral vectors in mice is mediated by dendritic cells and macrophages

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Lifelong elimination of hyperbilirubinemia in the Gunn rat with a single injection of helper-dependent adenoviral vector

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In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector

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Efficient production of human FVIII in hemophilic mice using lentiviral vectors

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Long-term lowering of plasma cholesterol levels in LDL-receptor-deficient WHHL rabbits by gene therapy

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Transduction of liver cells by lentiviral vectors: analysis in living animals by fluorescence imaging.

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Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice

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

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Long-term correction of bilirubin UDPglucuronyltransferase deficiency in rats by in utero lentiviral gene transfer

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Therapeutic lentivirus-mediated neonatal in vivo gene therapy in hyperbilirubinemic Gunn rats

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Gene therapy for organ grafts using rapid injection of naked DNA: application to the rat liver

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7 January 2021

Production of human clotting Factor IX without toxicity in mice after vascular delivery of a lentiviral vector

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Lentiviruses as gene transfer agents for delivery to non-dividing cells

1 reference

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7 January 2021

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

1 reference

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Dangerous liaisons: the role of "danger" signals in the immune response to gene therapy

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Activation of innate immunity in nonhuman primates following intraportal administration of adenoviral vectors.

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7 January 2021

Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors

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7 January 2021

Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors

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7 January 2021

Genetic lesions of bilirubin uridine-diphosphoglucuronate glucuronosyltransferase (UGT1A1) causing Crigler-Najjar and Gilbert syndromes: correlation of genotype to phenotype

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7 January 2021

An albumin enhancer located 10 kb upstream functions along with its promoter to direct efficient, liver-specific expression in transgenic mice.

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7 January 2021

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

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7 January 2021

Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow

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7 January 2021

Oncoretrovirus and lentivirus vectors pseudotyped with lymphocytic choriomeningitis virus glycoprotein: generation, concentration, and broad host range

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Large-scale production of pseudotyped lentiviral vectors using baculovirus GP64.

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Continuous high-titer HIV-1 vector production.

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Regulatory considerations for novel gene therapy products: a review of the process leading to the first clinical lentiviral vector

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Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences

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A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine

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Immunochemical analysis of uridine diphosphate-glucuronosyltransferase in four patients with the Crigler-Najjar syndrome type I.

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Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors.

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A third-generation lentivirus vector with a conditional packaging system

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inferred from DOI database lookup

Identifiers

10.1016/J.YMTHE.2005.09.022

1 reference

Europe PubMed Central

PubMed publication ID

19 September 2017

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

19 September 2017

ResearchGate publication ID

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