(Q34345151)

English

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

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scholarly article

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

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

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

molecular chaperones

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1 reference

based on heuristic

inferred from title

author name string

Richie Khanna

1

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

John J Flanagan

2

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

Jessie Feng

3

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

Rebecca Soska

4

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

Michelle Frascella

5

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

Lee J Pellegrino

6

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

Yi Lun

7

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

Darlene Guillen

8

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

David J Lockhart

9

1 reference

Europe PubMed Central

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1 August 2017

Kenneth J Valenzano

10

1 reference

Europe PubMed Central

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1 August 2017

language of work or name

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publication date

18 July 2012

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Europe PubMed Central

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1 August 2017

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

7

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

7

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

e40776

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

copyright license

Creative Commons Attribution 4.0 International

18 July 2012

1 reference

April 2022 Public Data File from Crossref

copyright status

0 references

Co-administration with the pharmacological chaperone AT1001 increases recombinant human α-galactosidase A tissue uptake and improves substrate reduction in Fabry mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Identification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

A randomized study of alglucosidase alfa in late-onset Pompe's disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Immortalization of murine muscle cells from lysosomal alpha-glucosidase deficient mice: a new tool to study pathophysiology and assess therapeutic strategies for Pompe disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Enzyme replacement therapy with alglucosidase alfa in 44 patients with late-onset glycogen storage disease type 2: 12-month results of an observational clinical trial.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Neural deficits contribute to respiratory insufficiency in Pompe disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Glycoengineered acid alpha-glucosidase with improved efficacy at correcting the metabolic aberrations and motor function deficits in a mouse model of Pompe disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Therapeutic approaches in glycogen storage disease type II/Pompe Disease.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Pompe's disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Isofagomine increases lysosomal delivery of exogenous glucocerebrosidase

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

A counterintuitive approach to treat enzyme deficiencies: use of enzyme inhibitors for restoring mutant enzyme activity

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

The use of differential scanning fluorimetry to detect ligand interactions that promote protein stability

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Pharmacological enhancement of mutated alpha-glucosidase activity in fibroblasts from patients with Pompe disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Characterization of pre- and post-treatment pathology after enzyme replacement therapy for Pompe disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Autophagy and lysosomes in Pompe disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

High-resolution light microscopy (HRLM) and digital analysis of Pompe disease pathology

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Pompe disease in infants and children

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

The natural course of infantile Pompe's disease: 20 original cases compared with 133 cases from the literature

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Generalized glycogen storage and cardiomegaly in a knockout mouse model of Pompe disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Structural and functional changes of lysosomal acid alpha-glucosidase during intracellular transport and maturation

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3399870

6 July 2018

Recombinant human alpha-glucosidase from rabbit milk in Pompe patients.

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0040776

21 January 2018

Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease.

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0040776

21 January 2018

Reversal of cardiac dysfunction after enzyme replacement in patients with infantile-onset Pompe disease.

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0040776

21 January 2018

Enzyme replacement therapy in the mouse model of Pompe disease

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0040776

21 January 2018

Abnormal mannose-6-phosphate receptor trafficking impairs recombinant alpha-glucosidase uptake in Pompe disease fibroblasts

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0040776

21 January 2018

The pharmacological chaperone 1-deoxynojirimycin increases the activity and lysosomal trafficking of multiple mutant forms of acid alpha-glucosidase

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0040776

21 January 2018

Both type 1 and type 2a muscle fibers can respond to enzyme therapy in Pompe disease.

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0040776

21 January 2018

Induction of tolerance to a recombinant human enzyme, acid alpha-glucosidase, in enzyme deficient knockout mice

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0040776

21 January 2018

Elimination of antibodies to recombinant enzyme in Pompe's disease.

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0040776

21 January 2018

Immune tolerance induction to enzyme-replacement therapy by co-administration of short-term, low-dose methotrexate in a murine Pompe disease model

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0040776

21 January 2018

Lentiviral gene therapy of murine hematopoietic stem cells ameliorates the Pompe disease phenotype

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0040776

21 January 2018

Promoter-specific lentivectors for long-term, cardiac-directed therapy of Fabry disease

1 reference

https://api.crossref.org/works/10.1371%2FJOURNAL.PONE.0040776

21 January 2018

Chemical chaperones improve transport and enhance stability of mutant alpha-glucosidases in glycogen storage disease type II

1 reference

https://pubmed.ncbi.nlm.nih.gov/22815812

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/22815812

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Synergy between the pharmacological chaperone 1-deoxygalactonojirimycin and the human recombinant alpha-galactosidase A in cultured fibroblasts from patients with Fabry disease

1 reference

https://pubmed.ncbi.nlm.nih.gov/22815812

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Uptake and stability of human and bovine acid α-glucosidase in cultured fibroblasts and skeletal muscle cells from glycogenosis type II patients

1 reference

https://pubmed.ncbi.nlm.nih.gov/22815812

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Antigen stability controls antigen presentation

1 reference

https://pubmed.ncbi.nlm.nih.gov/22815812

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1371/JOURNAL.PONE.0040776

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

PMC publication ID

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

1 August 2017

ResearchGate publication ID

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