(Q34763584)

4 August 2017

title

B-Cell Depletion is Protective Against Anti-AAV Capsid Immune Response: A Human Subject Case Study (English)

1 reference

human subject research project

4 August 2017

main subject

0 references

author

Sushrusha Nayak

6

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

author name string

M Corti

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

Me Elder

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

Dj Falk

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

L Lawson

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

Bk Smith

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

Tj Conlon

7

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

N Clément

8

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

K Erger

9

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

E Lavassani

10

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

M Green

11

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

Pa Doerfler

12

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

Rw Herzog

13

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

Bj Byrne

14

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

publication date

1 January 2014

1 reference

Molecular Therapy. Methods & Clinical Development

4 August 2017

published in

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

volume

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial

12 June 2020

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7 July 2018

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Algorithm for the early diagnosis and treatment of patients with cross reactive immunologic material-negative classic infantile pompe disease: a step towards improving the efficacy of ERT.

7 July 2018

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Humoral immunity to AAV vectors in gene therapy: challenges and potential solutions

7 July 2018

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A largely random AAV integration profile after LPLD gene therapy

7 July 2018

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B-Cell depletion and immunomodulation before initiation of enzyme replacement therapy blocks the immune response to acid alpha-glucosidase in infantile-onset Pompe disease.

7 July 2018

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Immune responses to AAV vectors: overcoming barriers to successful gene therapy

7 July 2018

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Phase I/II trial of adeno-associated virus-mediated alpha-glucosidase gene therapy to the diaphragm for chronic respiratory failure in Pompe disease: initial safety and ventilatory outcomes

7 July 2018

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Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review

7 July 2018

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Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue

7 July 2018

1 reference

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Proteasome inhibition is partially effective in attenuating pre-existing immunity against recombinant adeno-associated viral vectors

7 July 2018

1 reference

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Mapping the T helper cell response to acid α-glucosidase in Pompe mice

7 July 2018

1 reference

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Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: need for agents to target antibody-secreting plasma cells

7 July 2018

1 reference

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Systemic immunoregulatory and proteogenomic effects of tacrolimus to sirolimus conversion in liver transplant recipients

7 July 2018

1 reference

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Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4275004

Capsid-specific T-cell responses to natural infections with adeno-associated viruses in humans differ from those of nonhuman primates

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4275004

Induction of tolerance to factor VIII by transient co-administration with rapamycin.

7 July 2018

1 reference

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Pompe disease gene therapy.

7 July 2018

1 reference

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Rituximab: mechanism of action

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4275004

Progress and prospects: immune responses to viral vectors

7 July 2018

1 reference

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Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4275004

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT 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=4275004

Vision 1 year after gene therapy for Leber's congenital amaurosis

7 July 2018

1 reference

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Prophylactic immune tolerance induced by changing the ratio of antigen-specific effector to regulatory T cells

7 July 2018

1 reference

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Neural deficits contribute to respiratory insufficiency in Pompe disease

7 July 2018

1 reference

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Relative persistence of AAV serotype 1 vector genomes in dystrophic muscle

7 July 2018

1 reference

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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

7 July 2018

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Temporal neuropathologic and behavioral phenotype of 6neo/6neo Pompe disease mice

7 July 2018

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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

7 July 2018

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Pompe disease (glycogen storage disease type II): clinical features and enzyme replacement therapy.

7 July 2018

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7 July 2018

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

7 July 2018

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7 July 2018

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SBE-TAGS: an array-based method for efficient single-nucleotide polymorphism genotyping

7 July 2018

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Respiratory insufficiency in adult-type acid maltase deficiency

7 July 2018

1 reference

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Tooth bleaching induces changes in the vascular permeability of rat incisor pulps.

7 July 2018

1 reference

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GMFM in infancy: age-specific limitations and adaptations.

26 October 2018

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26 October 2018

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Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins

26 October 2018

1 reference

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26 October 2018

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Rituximab and immune tolerance in severe hemophilia A: a consecutive national cohort.

26 October 2018

1 reference

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Rituximab as a single agent in the management of adult patients with haemophilia A and inhibitors: marked reduction in inhibitor level and clinical improvement in bleeding but failure to eradicate the inhibitor

26 October 2018

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Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D.

26 October 2018

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CD8(+) T-cell responses to adeno-associated virus capsid in humans

26 October 2018

1 reference

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Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults

26 October 2018

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Pompe disease: Design, methodology, and early findings from the Pompe Registry

26 October 2018

1 reference

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Progress in medical genetics: map-based gene discovery and the molecular pathology of skeletal dysplasias

26 October 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25541616

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Molecular analysis and protein processing in late-onset Pompe disease patients with low levels of acid α-glucosidase activity

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25541616

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Case 15-2009: a man with coma after cardiac arrest

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/25541616

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1038/MTM.2014.33

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json

12 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25541616%20AND%20SRC:MED&resulttype=core&format=json