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Gene therapy for the hemophilias
scientific article
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instance of
scholarly article
1 reference
stated in
Europe PubMed Central
PubMed ID
10468539
retrieved
28 July 2017
review article
1 reference
stated in
Europe PubMed Central
title
Gene therapy for the hemophilias
(English)
1 reference
stated in
Europe PubMed Central
PubMed ID
10468539
retrieved
28 July 2017
main subject
gene therapy
0 references
hemophilia
0 references
author name string
M A Kay
series ordinal
1
1 reference
stated in
Europe PubMed Central
PubMed ID
10468539
retrieved
28 July 2017
K High
series ordinal
2
1 reference
stated in
Europe PubMed Central
PubMed ID
10468539
retrieved
28 July 2017
language of work or name
English
0 references
publication date
1 August 1999
1 reference
stated in
Europe PubMed Central
PubMed ID
10468539
retrieved
28 July 2017
published in
Proceedings of the National Academy of Sciences of the United States of America
1 reference
stated in
Europe PubMed Central
PubMed ID
10468539
retrieved
28 July 2017
volume
96
1 reference
stated in
Europe PubMed Central
PubMed ID
10468539
retrieved
28 July 2017
page(s)
9973-9975
1 reference
stated in
Europe PubMed Central
PubMed ID
10468539
retrieved
28 July 2017
issue
18
1 reference
stated in
Europe PubMed Central
PubMed ID
10468539
retrieved
28 July 2017
cites work
Christmas disease: a condition previously mistaken for haemophilia
1 reference
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PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
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21 June 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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PubMed Central
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21 June 2018
Implication of interfering antibody formation and apoptosis as two different mechanisms leading to variable duration of adenovirus-mediated transgene expression in immune-competent mice
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PubMed Central
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21 June 2018
Sustained correction of bleeding disorder in hemophilia B mice by gene therapy
1 reference
stated in
PubMed Central
reference URL
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21 June 2018
Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A
1 reference
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PubMed Central
reference URL
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21 June 2018
Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors
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PubMed Central
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21 June 2018
A coagulation factor IX-deficient mouse model for human hemophilia B.
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PubMed Central
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21 June 2018
A factor IX-deficient mouse model for hemophilia B gene therapy
1 reference
stated in
PubMed Central
reference URL
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21 June 2018
Haemophilia prophylaxis in young patients--a long-term follow-up
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PubMed Central
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21 June 2018
Proliferation induced by keratinocyte growth factor enhances in vivo retroviral-mediated gene transfer to mouse hepatocytes.
1 reference
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PubMed Central
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21 June 2018
A deletion mutation causes hemophilia B in Lhasa Apso dogs
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PubMed Central
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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
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21 June 2018
Antigen presentation--recent developments
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PubMed Central
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21 June 2018
Pseudotransduction of hepatocytes by using concentrated pseudotyped vesicular stomatitis virus G glycoprotein (VSV-G)-Moloney murine leukemia virus-derived retrovirus vectors: comparison of VSV-G and amphotropic vectors for hepatic gene transfer
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PubMed Central
reference URL
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21 June 2018
Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells
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PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
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21 June 2018
Sequences in the coding region of clotting factor VIII act as dominant inhibitors of RNA accumulation and protein production.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
retrieved
21 June 2018
Inversions disrupting the factor VIII gene are a common cause of severe haemophilia A
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
retrieved
21 June 2018
In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
retrieved
21 June 2018
In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
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21 June 2018
Tolerance, danger, and the extended family
1 reference
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PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
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21 June 2018
A general method for the generation of high-titer, pantropic retroviral vectors: highly efficient infection of primary hepatocytes
1 reference
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PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
retrieved
21 June 2018
Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
retrieved
21 June 2018
Why do so many haemophilia A patients develop an inhibitor?
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
retrieved
21 June 2018
Long-term hepatic adenovirus-mediated gene expression in mice following CTLA4Ig administration
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
retrieved
21 June 2018
Production of human factor IX in animals by genetically modified skin fibroblasts: potential therapy for hemophilia B.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
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21 June 2018
Canine hemophilia B resulting from a point mutation with unusual consequences
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
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21 June 2018
Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
retrieved
21 June 2018
Expression of human factor IX in rat capillary endothelial cells: toward somatic gene therapy for hemophilia B
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
retrieved
21 June 2018
Haemophilia B mutations in a complete Swedish population sample: a test of new strategy for the genetic counselling of diseases with high mutational heterogeneity
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PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
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21 June 2018
Genetics and molecular biology of haemophilias A and B
1 reference
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PubMed Central
reference URL
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21 June 2018
Expression of human factor IX in mice after injection of genetically modified myoblasts
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
retrieved
21 June 2018
Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A.
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1073%2FPNAS.96.18.9973
retrieved
21 January 2018
Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector
1 reference
stated in
Crossref
reference URL
https://api.crossref.org/works/10.1073%2FPNAS.96.18.9973
retrieved
21 January 2018
Plasma thromboplastin component (PTC) deficiency; a new disease resembling hemophilia
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
retrieved
30 October 2018
Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
retrieved
30 October 2018
Targeted inactivation of the coagulation factor IX gene causes hemophilia B in mice.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
retrieved
30 October 2018
Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
retrieved
30 October 2018
Efficient in vivo transduction of the neonatal mouse liver with pseudotyped retroviral vectors.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717
retrieved
30 October 2018
Hepatocyte growth factor induces hepatocyte proliferation in vivo and allows for efficient retroviral-mediated gene transfer in mice
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/10468539
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
The canine factor VIII cDNA and 5' flanking sequence
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/10468539
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
Development of factor VIII antibody in haemophilic monozygotic twins. European Study Group of Factor VIII Antibody
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/10468539
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
Identifiers
DOI
10.1073/PNAS.96.18.9973
1 reference
stated in
Europe PubMed Central
PubMed ID
10468539
retrieved
28 July 2017
ADS bibcode
1999PNAS...96.9973K
0 references
PMCID
33717
1 reference
stated in
Europe PubMed Central
PubMed ID
10468539
retrieved
28 July 2017
PubMed ID
10468539
1 reference
stated in
Europe PubMed Central
PubMed ID
10468539
retrieved
28 July 2017
ResearchGate publication ID
12830560
0 references
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