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Article

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

Molecular therapy : the journal of the American Society of Gene Therapy - 7 Mar 2018

Marshall Michael S, Issa Yazan, Jakubauskas Benas, Stoskute Monika, Elackattu Vince, Marshall Jeffrey N, Bogue Wil, Nguyen Duc, Hauck Zane, Rue Emily, Karumuthil-Melethil Subha, Zaric Violeta, Bosland Maarten, van Breemen Richard B, Givogri Maria I, Gray Steven J, Crocker Stephen J, Bongarzone Ernesto R

Abstract excerpt

We report a global adeno-associated virus (AAV)9-based gene therapy protocol to deliver therapeutic galactosylceramidase (GALC), a lysosomal enzyme that is deficient in Krabbe's disease. When globally administered via intrathecal, intracranial, and intravenous injections to newborn mice affected with GALC deficiency (twitcher mice), this approach largely surpassed prior published benchmarks of survival and...

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Identifiers

PMID: 29433937
DOI: 10.1016/j.ymthe.2018.01.009
PMCID: PMC5910889

Genes

GALC

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe's Disease after Global Gene Therapy.PMID 29433937Read ↗

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