Neonatal helper-dependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII (Q34550081)

3 August 2017

Neonatal helper-dependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII (English)

1 reference

3 August 2017

0 references

0 references

Coagulation factor VIII

0 references

0 references

1 reference

Chuhong Hu

1

1 reference

3 August 2017

Racel G Cela

2

1 reference

3 August 2017

Masataka Suzuki

3

1 reference

3 August 2017

Brendan Lee

4

1 reference

3 August 2017

Gerald S Lipshutz

5

1 reference

3 August 2017

language of work or name

English

0 references

publication date

18 January 2011

1 reference

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

3 August 2017

1 reference

3 August 2017

108

1 reference

3 August 2017

5

1 reference

3 August 2017

2082-2087

1 reference

Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gammac overexpression

3 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Neonatal gene transfer using lentiviral vector for murine Pompe disease: long-term expression and glycogen reduction

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Inhibitors of factor VIII in black patients with hemophilia

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Gene delivery to the juvenile mouse liver using AAV2/8 vectors

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Immunology of neonatal gene transfer

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Fetal and neonatal gene therapy: benefits and pitfalls

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Physical and infectious titers of helper-dependent adenoviral vectors: a method of direct comparison to the adenovirus reference material

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Pathogenic antibodies to coagulation factors. Part one: factor VIII and factor IX.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Environmental and genetic factors influencing inhibitor development

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Improved system for helper-dependent adenoviral vector production

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

The incidence of inhibitor development according to specific mutations - and treatment?

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

The epidemiology of inhibitors in haemophilia A: a systematic review

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Paediatric care of the child with haemophilia

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Preparation of helper-dependent adenoviral vectors

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Incidence of factor VIII inhibitors in severe haemophilia: the importance of patient age.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Activation of innate immunity in nonhuman primates following intraportal administration of adenoviral vectors.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Persistence of physiological self antigen is required for the regulation of self tolerance

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Adenoviral gene therapy leads to rapid induction of multiple chemokines and acute neutrophil-dependent hepatic injury in vivo

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Inhibitor antibody development and T cell response to human factor VIII in murine hemophilia A.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Long term correction of bilirubin-UDP-glucuronosyltransferase deficiency in Gunn rats by administration of a recombinant adenovirus during the neonatal period

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Further characterization of factor VIII-deficient mice created by gene targeting: RNA and protein studies

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Clonal anergy blocks in vivo growth of mature T cells and can be reversed in the absence of antigen

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. Safety, efficacy, and development of inhibitors. Kogenate Previously Untreated Patient Study Group

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Bleeding time in laboratory animals. II - A comparison of different assay conditions in rats

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Persistent expression of hF.IX After tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice

7 July 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Correction of murine hemophilia A and immunological differences of factor VIII variants delivered by helper-dependent adenoviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Factor VIII intron-1 inversion: frequency and inhibitor prevalence

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Inhibitors occur more frequently in African-American and Latino haemophiliacs

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Induction of tolerance to human factor VIII in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Partial correction of murine hemophilia A with neo-antigenic murine factor VIII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Sustained human factor VIII expression in hemophilia A mice following systemic delivery of a gutless adenoviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Acute toxicity after high-dose systemic injection of helper-dependent adenoviral vectors into nonhuman primates.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Increased hepatic transduction with reduced systemic dissemination and proinflammatory cytokines following hydrodynamic injection of helper-dependent adenoviral vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.1015571108

Absolute annual incidences of first events of venous thromboembolism and arterial vascular events in individuals with elevated FVIII:c. A prospective family cohort study.

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

On the mechanism of immunologic self recognition.

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

High doses of a helper-dependent adenoviral vector yield supraphysiological levels of alpha1-antitrypsin with negligible toxicity

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B.

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3033287

Role of clotting factor VIII in effect of von Willebrand factor on occurrence of deep-vein thrombosis

27 October 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21245323

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Haemophilia A: mutation type determines risk of inhibitor formation

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21245323

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

High plasma concentration of factor VIIIc is a major risk factor for venous thromboembolism

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/21245323

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1073/PNAS.1015571108

1 reference

3 August 2017

0 references

1 reference

3 August 2017

1 reference