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The gene therapy journey for hemophilia: are we there yet?
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title
The gene therapy journey for hemophilia: are we there yet?
(English)
1 reference
stated in
PubMed
main subject
gene therapy
0 references
hemophilia
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author name string
K. A. High
series ordinal
1
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stated in
Crossref
language of work or name
English
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stated in
PubMed
publication date
29 November 2012
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stated in
PubMed
published in
Blood
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stated in
PubMed
volume
120
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PubMed
issue
23
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PubMed
page(s)
4482-7
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PubMed
cites work
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
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Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial
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7 April 2017
Safety and efficacy of gene transfer for Leber's congenital amaurosis
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7 April 2017
Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis
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7 April 2017
In vivo genome editing restores haemostasis in a mouse model of haemophilia
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7 April 2017
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
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7 April 2017
Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates
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7 April 2017
Effect of gene therapy on visual function in Leber's congenital amaurosis
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7 April 2017
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
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7 April 2017
Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue
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28 September 2017
Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B.
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PubMed Central
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28 September 2017
Adenovirus-associated virus vector-mediated gene transfer in hemophilia B
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PubMed Central
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28 September 2017
Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients.
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PubMed Central
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28 September 2017
Adeno-associated virus antibody profiles in newborns, children, and adolescents
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28 September 2017
Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptide
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PubMed Central
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28 September 2017
A 10 patient case report on the impact of plasmapheresis upon neutralizing factors against adeno-associated virus (AAV) types 1, 2, 6, and 8.
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28 September 2017
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
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28 September 2017
Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial
1 reference
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PubMed Central
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28 September 2017
Assessing the potential for AAV vector genotoxicity in a murine model
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28 September 2017
Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors
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PubMed Central
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28 September 2017
In vivo efficacy of platelet-delivered, high specific activity factor VIII variants
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PubMed Central
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28 September 2017
Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.
1 reference
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PubMed Central
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28 September 2017
X-linked thrombophilia with a mutant factor IX (factor IX Padua).
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PubMed Central
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28 September 2017
Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors
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28 September 2017
Host and vector-dependent effects on the risk of germline transmission of AAV vectors
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PubMed Central
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28 September 2017
Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX.
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PubMed Central
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28 September 2017
Manufacturing and characterizing AAV-based vectors for use in clinical studies
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PubMed Central
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28 September 2017
Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates
1 reference
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PubMed Central
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28 September 2017
Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer
1 reference
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PubMed Central
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28 September 2017
Quantification of adeno-associated virus particles and empty capsids by optical density measurement
1 reference
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PubMed Central
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28 September 2017
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe 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=3512227
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28 September 2017
Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo
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=3512227
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28 September 2017
Sustained expression of human factor VIII in mice using a parvovirus-based vector.
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=3512227
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28 September 2017
Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein
1 reference
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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=3512227
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28 September 2017
Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver
1 reference
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PubMed Central
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28 September 2017
Adeno-associated virus vector integration junctions.
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PubMed Central
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28 September 2017
A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of Hemophilia A.
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=3512227
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31 May 2018
Alternative strategies for gene therapy of 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=3512227
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31 May 2018
Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies
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=3512227
retrieved
31 May 2018
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.
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=3512227
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31 May 2018
Activated factor VII: my story
1 reference
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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=3512227
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28 November 2018
Codon optimization of human factor VIII cDNAs leads to high-level expression
1 reference
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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=3512227
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28 November 2018
Gene therapy for immunodeficiency due to adenosine deaminase deficiency.
1 reference
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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3512227
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28 November 2018
Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses.
1 reference
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PubMed Central
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28 November 2018
AAV vector integration sites in mouse hepatocellular carcinoma.
1 reference
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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=3512227
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28 November 2018
CD8(+) T-cell responses to adeno-associated virus capsid in humans
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28 November 2018
In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promotes vector clearance.
1 reference
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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=3512227
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28 November 2018
Inadvertent germline transmission of AAV2 vector: findings in a rabbit model correlate with those in a human clinical trial
1 reference
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PubMed Central
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28 November 2018
Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs
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=3512227
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28 November 2018
Factor VIII ectopically expressed in platelets: efficacy in hemophilia A treatment
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=3512227
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28 November 2018
Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy
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=3512227
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28 November 2018
Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males
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=3512227
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28 November 2018
Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.
1 reference
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28 November 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
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3512227
retrieved
28 November 2018
Trial halted after gene shows up in semen
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/22829631
retrieved
12 December 2020
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inferred from PubMed ID database lookup
Identifiers
DOI
10.1182/BLOOD-2012-05-423210
1 reference
stated in
Consolidated OpenCitations Corpus – April 2017
OpenCitations bibliographic resource ID
3497371
OpenCitations bibliographic resource ID
3497371
1 reference
stated in
Consolidated OpenCitations Corpus – April 2017
OpenCitations bibliographic resource ID
3497371
PMC publication ID
3512227
1 reference
stated in
Consolidated OpenCitations Corpus – April 2017
OpenCitations bibliographic resource ID
3497371
PubMed publication ID
22829631
1 reference
stated in
Consolidated OpenCitations Corpus – April 2017
OpenCitations bibliographic resource ID
3497371
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