AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice (Q36203308)

12 August 2017

AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice (English)

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12 August 2017

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C Hu

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12 August 2017

G S Lipshutz

series ordinal

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12 August 2017

language of work or name

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12 January 2012

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12 August 2017

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1166-1176

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12 August 2017

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https://scigraph.springernature.com/pub.10.1038/gt.2011.200

12 August 2017

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cites work

AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta)

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

1 October 2017

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Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors

1 October 2017

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Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy

1 October 2017

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RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy

1 October 2017

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Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction

1 October 2017

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RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

1 October 2017

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Long-term rescue of a lethal murine model of methylmalonic acidemia using adeno-associated viral gene therapy

1 October 2017

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Systematic evaluation of AAV vectors for liver directed gene transfer in murine models.

1 October 2017

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Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model

1 October 2017

1 reference

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

1 October 2017

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Evaluation of gene promoters for liver expression by hydrodynamic gene transfer

1 October 2017

1 reference

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

1 October 2017

1 reference

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Existence of transient functional double-stranded DNA intermediates during recombinant AAV transduction

1 October 2017

1 reference

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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Liver transduction with recombinant adeno-associated virus is primarily restricted by capsid serotype not vector genotype

1 October 2017

1 reference

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Adeno-associated virus-based gene therapy for inherited disorders

1 October 2017

1 reference

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Donor-specific B-cell tolerance after ABO-incompatible infant heart transplantation

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

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

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

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

1 October 2017

1 reference

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Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

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

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

The role of hepatocytes and oval cells in liver regeneration and repopulation

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Rate limiting steps of AAV transduction and implications for human gene therapy.

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Cellular immune responses in neonates

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Advances toward gene therapy for hemophilia at the millennium

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

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 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Gene therapy for the hemophilias

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Adeno-associated virus vectors can be efficiently produced without helper virus

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Recombinant adeno-associated virus mediates a high level of gene transfer but less efficient integration in the K562 human hematopoietic cell line

1 October 2017

1 reference

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

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Effective clearance of a persistent viral infection requires cooperation between virus-specific Lyt2+ T cells and nonspecific bone marrow-derived cells

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

T-cell tolerance: exposure to virus in utero does not cause a permanent deletion of specific T cells

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Hemophilia gene therapy: a Holy Grail found

1 October 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

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

2 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

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

2 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

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

2 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Efficient production of recombinant human factor VIII by co-expression of the heavy and light chains

2 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

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

2 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors

2 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses.

22 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

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

22 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Long-term efficacy of adeno-associated virus serotypes 8 and 9 in hemophilia a dogs and mice

22 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs

22 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector

22 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

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

22 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Neonatal gene transfer with a retroviral vector results in tolerance to human factor IX in mice and dogs

22 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Phenotypic correction of a mouse model of hemophilia A using AAV2 vectors encoding the heavy and light chains of FVIII.

22 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector

22 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Dual vectors expressing murine factor VIII result in sustained correction of hemophilia A mice

22 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Cell proliferation and renewal of normal hepatocytes and bile duct cells in adult mouse liver

22 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

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

22 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

Metabolic instability of plasmid DNA in the cytosol: a potential barrier to gene transfer

22 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3432168

The appearance of immunological competence at an early age in New Zealand black mice

22 September 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/22241178

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Response of human foetal thymocytes to phytohaemagglutinin (PHA)

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/22241178

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

The kinetics of rAAV integration in the liver

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/22241178

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/GT.2011.200

1 reference

Dimensions Publication ID

12 August 2017

0 references

1 reference

12 August 2017

1 reference