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

Extended Normal Life After AAVrh10-mediated Gene Therapy in the Mouse Model of Krabbe Disease

31 Jul 2012

Abstract excerpt

Globoid cell leukodystrophy (GLD) or Krabbe disease is a neurodegenerative disorder caused by the deficiency of the lysosomal enzyme galactocerebrosidase (GALC). This deficiency results in accumulation of certain galactolipids including psychosine which is cytotoxic for myelin-producing cells. Treatment of human patients at this time is limited to hematopoietic stem cell transplantation (HSCT) that appears to...

Read the complete abstract on PubMed

Topics

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

Share to Lysosomal Storage Disorders Research
Share to RNA regulation and disease
Share to Virus-based gene therapy research

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 22850681
DOI: 10.1038/mt.2012.153

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Extended Normal Life After AAVrh10-mediated Gene Therapy in the Mouse Model of Krabbe DiseasePMID 22850681Read ↗

Intrathecal administration of AAV/GALC vectors in 10–11‐day‐old twitcher mice improves survival and is enhanced by bone marrow transplantSemantic proximity Read ↗

AAV-Mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophySemantic proximity Read ↗

Single‐dose intracerebroventricular administration of galactocerebrosidase improves survival in a mouse model of globoid cell leukodystrophySemantic proximity Read ↗

The twitcher mouse: a model for Krabbe disease and for experimental therapies.Semantic proximity Read ↗

Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe diseaseSemantic proximity Read ↗

AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2.Semantic proximity Read ↗

Enzyme replacement therapy results in substantial improvements in early clinical phenotype in a mouse model of globoid cell leukodystrophy.Semantic proximity Read ↗

Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe's Disease after Global Gene Therapy.Semantic proximity Read ↗

An Engineered Galactosylceramidase Construct Improves AAV Gene Therapy for Krabbe Disease in Twitcher MiceSemantic proximity Read ↗

Significant correction of pathology in brains of twitcher mice following injection of genetically modified mouse neural progenitor cells.Semantic proximity Read ↗

Generation of a mouse with low galactocerebrosidase activity by gene targeting: a new model of globoid cell leukodystrophy (Krabbe disease).Semantic proximity Read ↗

Central Nervous System-directed AAV2/5-Mediated Gene Therapy Synergizes with Bone Marrow Transplantation in the Murine Model of Globoid-cell LeukodystrophySemantic proximity Read ↗

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