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Gene therapy for hemophilia
scientific article published on 01 January 2001
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Europe PubMed Central
PubMed publication ID
11269333
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:11269333%20AND%20SRC:MED&resulttype=core&format=json
retrieved
5 November 2019
review article
1 reference
stated in
Europe PubMed Central
title
Gene therapy for hemophilia
(English)
1 reference
stated in
Europe PubMed Central
PubMed publication ID
11269333
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:11269333%20AND%20SRC:MED&resulttype=core&format=json
retrieved
5 November 2019
main subject
hemophilia
1 reference
based on heuristic
inferred from title
author name string
Chuah MK
series ordinal
1
1 reference
stated in
Europe PubMed Central
PubMed publication ID
11269333
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:11269333%20AND%20SRC:MED&resulttype=core&format=json
retrieved
5 November 2019
Collen D
series ordinal
2
1 reference
stated in
Europe PubMed Central
PubMed publication ID
11269333
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:11269333%20AND%20SRC:MED&resulttype=core&format=json
retrieved
5 November 2019
VandenDriessche T
series ordinal
3
1 reference
stated in
Europe PubMed Central
PubMed publication ID
11269333
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:11269333%20AND%20SRC:MED&resulttype=core&format=json
retrieved
5 November 2019
language of work or name
English
0 references
publication date
1 January 2001
1 reference
stated in
Europe PubMed Central
PubMed publication ID
11269333
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:11269333%20AND%20SRC:MED&resulttype=core&format=json
retrieved
5 November 2019
published in
Journal of Gene Medicine
1 reference
stated in
Europe PubMed Central
PubMed publication ID
11269333
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:11269333%20AND%20SRC:MED&resulttype=core&format=json
retrieved
5 November 2019
volume
3
1 reference
stated in
Europe PubMed Central
PubMed publication ID
11269333
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:11269333%20AND%20SRC:MED&resulttype=core&format=json
retrieved
5 November 2019
issue
1
1 reference
stated in
Europe PubMed Central
PubMed publication ID
11269333
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:11269333%20AND%20SRC:MED&resulttype=core&format=json
retrieved
5 November 2019
page(s)
3-20
1 reference
stated in
Europe PubMed Central
PubMed publication ID
11269333
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:11269333%20AND%20SRC:MED&resulttype=core&format=json
retrieved
5 November 2019
cites work
Advances toward gene therapy for hemophilia at the millennium
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Gene therapy and the hemophilias
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Human gene therapy
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Gene therapy for the haemophilias: current status.
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Deficiencies in Factors IX and VIII: What Is Now Known
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Gene therapy for hemophilia: hopes and hurdles
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Gene therapy for hemophilia: a step closer to reality
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Gene therapy for the hemophilias
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An alternative approach to somatic cell gene therapy
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Phenotypic correction of factor IX deficiency in skin fibroblasts of hemophilic dogs
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An ex vivo keratinocyte model for gene therapy of hemophilia B.
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Loss of expression of a retrovirus-transduced gene in human keratinocytes
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Development and analysis of retroviral vectors expressing human factor VIII as a potential gene therapy for hemophilia A.
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21 January 2018
Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice
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21 January 2018
Expression of human factor IX in rat capillary endothelial cells: toward somatic gene therapy for hemophilia B
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Expression of human factor IX in mice after injection of genetically modified myoblasts
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21 January 2018
Gene therapy via primary myoblasts: long-term expression of factor IX protein following transplantation in vivo
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21 January 2018
Retroviral-mediated in vivo gene transfer into muscle cells and synthesis of human factor IX in mice
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Expression of biologically active human factor IX in human hematopoietic cells after retroviral vector-mediated gene transduction
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Systemic delivery of human growth hormone or human factor IX in dogs by reintroduced genetically modified autologous bone marrow stromal cells
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21 January 2018
Expression of factor VIII by murine liver sinusoidal endothelial cells
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A factor IX-deficient mouse model for hemophilia B gene therapy
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Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A
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In vivo production of human factor VII in mice after intrasplenic implantation of primary fibroblasts transfected by receptor-mediated, adenovirus-augmented gene delivery
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Efficient transfection of primary cells in a canine hemophilia B model using adenovirus-polylysine-DNA complexes
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Characterization of the human factor VIII gene
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Expression of active human factor VIII from recombinant DNA clones
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Molecular cloning of a cDNA encoding human antihaemophilic factor
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A large region (approximately equal to 95 kDa) of human factor VIII is dispensable for in vitro procoagulant activity
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Sequences in the coding region of clotting factor VIII act as dominant inhibitors of RNA accumulation and protein production.
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The human clotting factor VIII cDNA contains an autonomously replicating sequence consensus- and matrix attachment region-like sequence that binds a nuclear factor, represses heterologous gene expression, and mediates the transcriptional effects of
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Sequences within the coding regions of clotting factor VIII and CFTR block transcriptional elongation
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The levels of endoplasmic reticulum proteins and ATP affect folding and secretion of selective proteins
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Use of retroviral vectors for gene transfer and expression
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A triploblast origin for Myxozoa?
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Safety Issues Related to Retroviral-Mediated Gene Transfer in Humans
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Report to the NIH Recombinant DNA Advisory Committee on murine replication-competent retrovirus (RCR) assays (February 17, 1993).
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Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer
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Splicing and the formation of stable RNA
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Toward gene therapy for hemophilia A: long-term persistence of factor VIII-secreting fibroblasts after transplantation into immunodeficient mice.
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Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A
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Long-term persistence of human bone marrow stromal cells transduced with factor VIII-retroviral vectors and transient production of therapeutic levels of human factor VIII in nonmyeloablated immunodeficient mice
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In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs
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Liver-directed gene therapy: quantitative evaluation of promoter elements by using in vivo retroviral transduction
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Proliferation induced by keratinocyte growth factor enhances in vivo retroviral-mediated gene transfer to mouse hepatocytes.
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Hepatocyte growth factor induces hepatocyte proliferation in vivo and allows for efficient retroviral-mediated gene transfer in mice
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21 January 2018
Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice
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A stable human-derived packaging cell line for production of high titer retrovirus/vesicular stomatitis virus G pseudotypes
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Sensitization of cells and retroviruses to human serum by (alpha 1-3) galactosyltransferase.
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21 January 2018
Liver stem cells: a two compartment system
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21 January 2018
Liver regeneration: prospects for therapy based on new technologies
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21 January 2018
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21 January 2018
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21 January 2018
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21 January 2018
Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences
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Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors
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21 January 2018
Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors
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Partial correction of murine hemophilia A with neo-antigenic murine factor VIII.
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Short-term correction of factor VIII deficiency in a murine model of hemophilia A after delivery of adenovirus murine factor VIII in utero
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21 January 2018
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A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase
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21 January 2018
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21 January 2018
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21 January 2018
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21 January 2018
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21 January 2018
Human factor VIII can be packaged and functionally expressed in an adeno-associated virus background: applicability to haemophilia A gene therapy
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21 January 2018
Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein
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21 January 2018
Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus
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21 January 2018
Long-term in vivo expression of retrovirus-mediated gene transfer in mouse fibroblast implants
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21 January 2018
Ex vivo fibroblast transduction in rabbits results in long-term (>600 days) factor IX expression in a small percentage of animals
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21 January 2018
Circulating human or canine factor IX from retrovirally transduced primary myoblasts and established myoblast cell lines grafted into murine skeletal muscle
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21 January 2018
In vivo retrovirus-mediated gene transfer into multiple hematopoietic lineages in rabbits without preconditioning
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21 January 2018
Expression of human factor IX in rabbit hepatocytes by retrovirus-mediated gene transfer: potential for gene therapy of hemophilia B.
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21 January 2018
Towards gene therapy for haemophilia B using primary human keratinocytes
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21 January 2018
Recombinant factor IX secreted by transduced human keratinocytes is biologically active
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21 January 2018
Gut epithelial cells as targets for gene therapy of hemophilia
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21 January 2018
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21 January 2018
Persistent delivery of factor IX in mice: gene therapy for hemophilia using implantable microcapsules
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21 January 2018
In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs
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21 January 2018
Adenovirus mediated expression of therapeutic plasma levels of human factor IX in mice
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21 January 2018
Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression
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21 January 2018
Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX.
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21 January 2018
Gene Therapy for Hemophilia B: Host Immunosuppression Prolongs the Therapeutic Effect of Adenovirus-Mediated Factor IX Expression
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21 January 2018
Persistent expression of human clotting factor IX from mouse liver after intravenous injection of adeno-associated virus vectors
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21 January 2018
Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors
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21 January 2018
Sustained correction of bleeding disorder in hemophilia B mice by gene therapy
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21 January 2018
Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver
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21 January 2018
Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia
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21 January 2018
Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector
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21 January 2018
Persistent expression of canine factor IX in hemophilia B canines
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21 January 2018
Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system
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21 January 2018
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21 January 2018
Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro
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21 January 2018
Correction of the UDP-glucuronosyltransferase gene defect in the gunn rat model of crigler-najjar syndrome type I with a chimeric oligonucleotide
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21 January 2018
In vivo site-directed mutagenesis of the factor IX gene by chimeric RNA/DNA oligonucleotides
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21 January 2018
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.
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An innate sense of danger
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21 January 2018
Identifiers
DOI
10.1002/1521-2254(200101/02)3:1<3::AID-JGM167>3.0.CO;2-H
0 references
PubMed publication ID
11269333
1 reference
stated in
Europe PubMed Central
PubMed publication ID
11269333
reference URL
https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:11269333%20AND%20SRC:MED&resulttype=core&format=json
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5 November 2019
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