Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade (Q34999729)

5 August 2017

Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade (English)

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5 August 2017

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Elizabeth D Brooks

3

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Herman Waldmann

object named as

Herman Waldmann

9

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Federico Mingozzi

8

object named as

Federico Mingozzi

1 reference

5 August 2017

Sang-oh Han

1

1 reference

5 August 2017

Songtao Li

2

1 reference

5 August 2017

Elisa Masat

4

1 reference

5 August 2017

Christian Leborgne

5

1 reference

5 August 2017

Suhrad Banugaria

6

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5 August 2017

Andrew Bird

7

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5 August 2017

Dwight Koeberl

10

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5 August 2017

1 January 2015

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5 August 2017

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1

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26-35

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describes a project that uses

5 August 2017

ImageJ

1 reference

11 June 2022

https://www.ebi.ac.uk/europepmc/webservices/rest/PMC4303018/fullTextXML

inferred from PubMed Central ID database lookup

cites work

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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.

8 July 2018

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Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease

8 July 2018

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Deficiency in MyD88 Signaling Results in Decreased Antibody Responses to an Adeno-Associated Virus Vector in Murine Pompe Disease

8 July 2018

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Pre-existing anti-adeno-associated virus antibodies as a challenge in AAV gene therapy

8 July 2018

1 reference

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Immune Modulation Therapy in a CRIM-Positive and IgG Antibody-Positive Infant with Pompe Disease Treated with Alglucosidase Alfa: A Case Report

8 July 2018

1 reference

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The pharmacological chaperone AT2220 increases recombinant human acid α-glucosidase uptake and glycogen reduction in a mouse model of Pompe disease

8 July 2018

1 reference

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Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B.

8 July 2018

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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

8 July 2018

1 reference

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Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease

8 July 2018

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Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease.

8 July 2018

1 reference

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Long-term efficacy following readministration of an adeno-associated virus vector in dogs with glycogen storage disease type Ia

8 July 2018

1 reference

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Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector

8 July 2018

1 reference

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Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine

8 July 2018

1 reference

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The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease

8 July 2018

1 reference

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Impact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors

8 July 2018

1 reference

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FcγRIV deletion reveals its central role for IgG2a and IgG2b activity in vivo

8 July 2018

1 reference

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Antibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe disease.

8 July 2018

1 reference

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Suppression of autophagy permits successful enzyme replacement therapy in a lysosomal storage disorder--murine Pompe disease

8 July 2018

1 reference

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Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia.

8 July 2018

1 reference

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Pompe disease in infants: improving the prognosis by newborn screening and early treatment.

8 July 2018

1 reference

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

8 July 2018

1 reference

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Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease

8 July 2018

1 reference

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Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus-mediated gene therapy

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Role of autophagy in the pathogenesis of Pompe disease.

8 July 2018

1 reference

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Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer

8 July 2018

1 reference

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Pre-existing AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes

8 July 2018

1 reference

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Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II.

8 July 2018

1 reference

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Anti-CD4 monoclonal antibody treatment in acute and early chronic antigen induced arthritis: influence on macrophage activation

8 July 2018

1 reference

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

8 July 2018

1 reference

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Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial

8 July 2018

1 reference

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Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase

8 July 2018

1 reference

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Transduction by adeno-associated virus vectors in the rabbit airway: efficiency, persistence, and readministration.

8 July 2018

1 reference

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Induction of tolerance by monoclonal antibody therapy

8 July 2018

1 reference

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Induction of classical transplantation tolerance in the adult

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Effects of immune modulation therapy in the first Croatian infant diagnosed with Pompe disease: a 3-year follow-up study.

8 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

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

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Muscle as a target for supplementary factor IX gene transfer

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

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

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Human immunoglobulin inhibits liver transduction by AAV vectors at low AAV2 neutralizing titers in SCID mice

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II.

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Major role of local immune responses in antibody formation to factor IX in AAV gene transfer

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage disease type II.

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Improved efficacy of gene therapy approaches for Pompe disease using a new, immune-deficient GSD-II mouse model

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Enzyme replacement therapy in the mouse model of Pompe disease

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Efficacy of gene therapy for a prototypical lysosomal storage disease (GSD-II) is critically dependent on vector dose, transgene promoter, and the tissues targeted for vector transduction

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Mechanisms in CD4 antibody-mediated transplantation tolerance: kinetics of induction, antigen dependency and role of regulatory T cells.

26 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4303018

Induction of Immunological Tolerance/Hyporesponsiveness in Baboons with a Nondepleting CD4 Antibody

26 October 2018

1 reference

12 December 2020

CD25+CD4+ regulatory T cells generated by exposure to a model protein antigen prevent allograft rejection: antigen-specific reactivation in vivo is critical for bystander regulation

1 reference

12 December 2020

High antibody titer in an adult with Pompe disease affects treatment with alglucosidase alfa

1 reference

12 December 2020

Enzyme replacement therapy induces T-cell responses in late-onset Pompe disease

1 reference

12 December 2020

Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease

1 reference

12 December 2020

Prevention of Diabetes but not Insulitis in NOD Mice Injected with Antibody to CD4

1 reference

12 December 2020

Identifiers

DOI

10.1089/HUM.2014.115

1 reference

5 August 2017

1 reference

5 August 2017

1 reference