Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

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

Transgenic research - 1 Apr 2003

Raben Nina, Nagaraju Kanneboyina, Lee Alicia, Lu Nina, Rivera Yesenia, Jatkar Tejas, Hopwood John J, Plotz Paul H

Abstract excerpt

When knockout mice are used to test the efficacy of recombinant human proteins, the animals often develop antibodies to the enzyme, precluding long-term pre-clinical studies. This has been a problem with a number of models, for example, the evaluation of gene or enzyme replacement therapies in a knockout model of glycogen storage disease type II (GSDII; Pompe syndrome). In this disease, the lack of acid...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to alpha-Glucosidases
Share to Autoantibodies

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to CHO Cells

Share to Cricetinae

Share to Glycogen Storage Disease Type II

Share to Mice, Transgenic

Share to Recombinant Proteins

Source

Identifiers

PMID: 12739885
DOI: 10.1023/a:1022998010833

Genes

GAA

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Induction of tolerance to a recombinant human enzyme, acid alpha-glucosidase, in enzyme deficient knockout mice.PMID 12739885Read ↗

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

Conditional tissue-specific expression of the acid alpha-glucosidase (GAA) gene in the GAA knockout mice: implications for therapySemantic proximity Read ↗

Partial phenotypic correction and immune tolerance induction to enzyme replacement therapy after hematopoietic stem cell gene transfer of alpha-glucosidase in Pompe disease.Semantic proximity Read ↗

Targeted approaches to induce immune tolerance for Pompe disease therapySemantic proximity Read ↗

Targeted delivery of acid alpha-glucosidase corrects skeletal muscle phenotypes in Pompe disease miceSemantic proximity Read ↗

Glycogen stored in skeletal but not in cardiac muscle in acid alpha-glucosidase mutant (Pompe) mice is highly resistant to transgene-encoded human enzyme.Semantic proximity Read ↗

Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.Semantic proximity Read ↗

Glycogen Stored in Skeletal but Not in Cardiac Muscle in Acid α-Glucosidase Mutant (Pompe) Mice Is Highly Resistant to Transgene-Encoded Human EnzymeSemantic proximity Read ↗

Modulation of disease severity in mice with targeted disruption of the acid alpha-glucosidase gene.Semantic proximity Read ↗

Advancements in AAV-mediated Gene Therapy for Pompe DiseaseSemantic proximity Read ↗

Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe diseaseSemantic proximity Read ↗

Acid alpha-glucosidase deficiency (glycogenosis type II, Pompe disease).Semantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.