Gene therapy for the hemophilias (Q33723486)

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scientific article

Language	Label	Description	Also known as
English	Gene therapy for the hemophilias	scientific article

Statements

scholarly article

1 reference

Europe PubMed Central

PubMed publication ID

28 July 2017

1 reference

Europe PubMed Central

Gene therapy for the hemophilias (English)

1 reference

Europe PubMed Central

PubMed publication ID

28 July 2017

0 references

0 references

author name string

M A Kay

1

1 reference

Europe PubMed Central

PubMed publication ID

28 July 2017

K High

2

1 reference

Europe PubMed Central

PubMed publication ID

28 July 2017

language of work or name

0 references

publication date

1 August 1999

1 reference

Europe PubMed Central

PubMed publication ID

28 July 2017

Proceedings of the National Academy of Sciences of the United States of America

1 reference

Europe PubMed Central

PubMed publication ID

28 July 2017

96

1 reference

Europe PubMed Central

PubMed publication ID

28 July 2017

18

1 reference

Europe PubMed Central

PubMed publication ID

28 July 2017

9973-9975

1 reference

Europe PubMed Central

PubMed publication ID

28 July 2017

Christmas disease: a condition previously mistaken for haemophilia

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Sustained correction of bleeding disorder in hemophilia B mice by gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

A coagulation factor IX-deficient mouse model for human hemophilia B

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

A factor IX-deficient mouse model for hemophilia B gene therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Haemophilia prophylaxis in young patients--a long-term follow-up

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

A deletion mutation causes hemophilia B in Lhasa Apso dogs

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Antigen presentation--recent developments

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Sequences in the coding region of clotting factor VIII act as dominant inhibitors of RNA accumulation and protein production.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Inversions disrupting the factor VIII gene are a common cause of severe haemophilia A

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Tolerance, danger, and the extended family

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

A general method for the generation of high-titer, pantropic retroviral vectors: highly efficient infection of primary hepatocytes

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Why do so many haemophilia A patients develop an inhibitor?

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Long-term hepatic adenovirus-mediated gene expression in mice following CTLA4Ig administration

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Production of human factor IX in animals by genetically modified skin fibroblasts: potential therapy for hemophilia B.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Canine hemophilia B resulting from a point mutation with unusual consequences

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Expression of human factor IX in rat capillary endothelial cells: toward somatic gene therapy for hemophilia B

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Genetics and molecular biology of haemophilias A and B

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

21 June 2018

Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.96.18.9973

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

https://api.crossref.org/works/10.1073%2FPNAS.96.18.9973

21 January 2018

Plasma thromboplastin component (PTC) deficiency; a new disease resembling hemophilia

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

30 October 2018

Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

30 October 2018

Targeted inactivation of the coagulation factor IX gene causes hemophilia B in mice.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

30 October 2018

Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

30 October 2018

Efficient in vivo transduction of the neonatal mouse liver with pseudotyped retroviral vectors.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=33717

30 October 2018

Hepatocyte growth factor induces hepatocyte proliferation in vivo and allows for efficient retroviral-mediated gene transfer in mice

1 reference

https://pubmed.ncbi.nlm.nih.gov/10468539

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

The canine factor VIII cDNA and 5' flanking sequence

1 reference

https://pubmed.ncbi.nlm.nih.gov/10468539

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

https://pubmed.ncbi.nlm.nih.gov/10468539

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1073/PNAS.96.18.9973

1 reference

Europe PubMed Central

PubMed publication ID

28 July 2017

1999PNAS...96.9973K

0 references

PMC publication ID

1 reference

Europe PubMed Central

PubMed publication ID

28 July 2017

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

28 July 2017

ResearchGate publication ID

0 references

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