Therapeutic levels of human protein C in rats after retroviral vector-mediated hepatic gene therapy (Q37383736)

20 August 2017

Therapeutic levels of human protein C in rats after retroviral vector-mediated hepatic gene therapy (English)

1 reference

20 August 2017

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S R Cai

1

1 reference

20 August 2017

S C Kennedy

2

1 reference

20 August 2017

W M Bowling

3

1 reference

20 August 2017

M W Flye

4

1 reference

20 August 2017

K P Ponder

5

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20 August 2017

language of work or name

English

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publication date

1 June 1998

1 reference

Journal of Clinical Investigation

20 August 2017

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20 August 2017

101

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20 August 2017

12

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20 August 2017

2831-2841

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Insulin-I131 metabolism in human subjects: demonstration of insulin binding globulin in the circulation of insulin treated subjects

20 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Replacement therapy with a monoclonal antibody purified protein C concentrate in newborns with severe congenital protein C deficiency

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Successful expression of human factor IX following repeat administration of adenoviral vector in mice.

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Enzyme therapy in Gaucher disease type 1: dosage efficacy and adverse effects in 33 patients treated for 6 to 24 months

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Gene transfer in vivo: sustained expression and regulation of genes introduced into the liver by receptor-targeted uptake

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Liver-directed gene therapy: quantitative evaluation of promoter elements by using in vivo retroviral transduction

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Ophthalmic manifestations of neonatal protein C deficiency

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Characterization of a cDNA coding for human protein C

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Severe inherited "homozygous" protein C deficiency in a newborn infant

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Murine sarcoma and leukemia viruses: assay using clonal lines of contact-inhibited mouse cells

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Neonatal purpura fulminans: a genetic disorder related to the absence of protein C in blood.

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Absence of thrombosis in subjects with heterozygous protein C deficiency

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

An alternative approach to somatic cell gene therapy

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Production of glycosylated physiologically "normal" human alpha 1-antitrypsin by mouse fibroblasts modified by insertion of a human alpha 1-antitrypsin cDNA using a retroviral vector

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

The human and rodent intestinal fatty acid binding protein genes. A comparative analysis of their structure, expression, and linkage relationships

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Improved retroviral vectors for gene transfer and expression

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Homozygous protein C deficiency: early treatment with warfarin

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Targeted disruption of the c-src proto-oncogene leads to osteopetrosis in mice

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Structural features in eukaryotic mRNAs that modulate the initiation of translation

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Expression of human factor IX in mice after injection of genetically modified myoblasts

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Gene therapy via primary myoblasts: long-term expression of factor IX protein following transplantation in vivo

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

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

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Prospects for gene therapy of haemophilia A and B.

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Enzyme therapy in Gaucher disease type 1: effect of neutralizing antibodies to acid beta-glucosidase

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Gene therapy and the hemophilias

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Therapeutic levels of functional human factor X in rats after retroviral-mediated hepatic gene therapy.

26 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

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

26 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Complete short-term correction of canine hemophilia A by in vivo gene therapy

26 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

A simple and efficient method for the concentration and purification of recombinant retrovirus for increased hepatocyte transduction in vivo

26 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs

26 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Improved methods of retroviral vector transduction and production for gene therapy

26 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

In vivo gene delivery and expression of physiological levels of functional human factor VIII in mice

26 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

The initial, rapid-phase disappearance of intravenous radioinsulin in diabetes.

26 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Treatment of pituitary dwarfism with methionyl human growth hormone in Japan

26 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Successful treatment of homozygous protein C deficiency by hepatic transplantation

26 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Construction and use of a safe and efficient amphotropic packaging cell line

26 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Liver-directed gene therapy: a retroviral vector with a complete LTR and the ApoE enhancer-alpha 1-antitrypsin promoter dramatically increases expression of human alpha 1-antitrypsin in vivo

26 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

High-level tissue-specific expression of functional human factor VIII in mice

2 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Antibodies to factor VIII in plasma of patients with hemophilia A and normal subjects

2 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Adenovirus mediated expression of therapeutic plasma levels of human factor IX in mice

2 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Quantitative evaluation of liver-specific promoters from retroviral vectors after in vivo transduction of hepatocytes

2 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Protein C deficiency in a controlled series of unselected outpatients: an infrequent but clear risk factor for venous thrombosis (Leiden Thrombophilia Study)

2 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=508874

Severe homozygous protein C deficiency

2 September 2018

1 reference

12 December 2020

Coumarin prophylaxis for fulminant purpura syndrome due to homozygous protein C deficiency

1 reference

12 December 2020

Homozygous protein C deficiency with moderately severe clinical symptoms

1 reference

12 December 2020

Purpura fulminans in a Chinese boy with congenital protein C deficiency

1 reference

12 December 2020

Persistent systemic production of human factor IX in mice by skeletal myoblast-mediated gene transfer: feasibility of repeat application to obtain therapeutic levels

1 reference

12 December 2020

The Effect of Insulin-binding Serum Globulin on Insulin Requirement

1 reference

12 December 2020

Protein C inhibitor: structure and function

1 reference

12 December 2020

A second plasma inhibitor of activated protein C: alpha 1-antitrypsin

1 reference

12 December 2020

Determination of plasma protein C inhibitor and of two activated protein C-inhibitor complexes in normals and in patients with intravascular coagulation and thrombotic disease

1 reference

12 December 2020

Evaluation of a new protein-C concentrate and comparison of protein-C assays in a child with congenital protein-C deficiency

1 reference

12 December 2020

Problems of oral anticoagulation in an adult with homozygous protein C deficiency and late onset of thrombosis

1 reference

12 December 2020

Normalization of markers of coagulation activation with a purified protein C concentrate in adults with homozygous protein C deficiency

1 reference

12 December 2020

Antibody response in patients with Gaucher disease after repeated infusion with macrophage-targeted glucocerebrosidase

1 reference

12 December 2020

Protein C deficiency in Kuwait

1 reference

12 December 2020

Homozygous protein C deficiency--management with protein C concentrate

1 reference

12 December 2020

Protein C deficiency: a database of mutations, 1995 update. On behalf of the Subcommittee on Plasma Coagulation Inhibitors of the Scientific and Standardization Committee of the ISTH

1 reference

12 December 2020

Progress towards gene therapy for the hemophilias

1 reference

12 December 2020

A review of mutations causing deficiencies of antithrombin, protein C and protein S

1 reference

12 December 2020

Treatment of homozygous protein C deficiency with subcutaneous protein C concentrate

1 reference

12 December 2020

Purpura fulminans in severe congenital protein C deficiency: monitoring of treatment with protein C concentrate

1 reference

12 December 2020

Species specificity of anticoagulant activity of activated human protein C: involvement of factor V as well as protein S

1 reference

12 December 2020

Insulin antibodies and the control of diabetes

1 reference

12 December 2020

Inherited protein C deficiency and coumarin-responsive chronic relapsing purpura fulminans in a newborn infant

1 reference

12 December 2020

Homozygous protein C deficiency manifested by massive venous thrombosis in the newborn

1 reference

12 December 2020

Identifiers

DOI

10.1172/JCI1880

1 reference

20 August 2017

1 reference

20 August 2017

1 reference