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Article

Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors.

The Journal of neuroscience : the official journal of the Society for Neuroscience - 12 Oct 2005

Liu Gumei, Martins Inês, Wemmie John A, Chiorini John A, Davidson Beverly L

Abstract excerpt

Lysosomal storage diseases (LSDs) represent a significant portion of inborn metabolic disorders. More than 60% of LSDs have CNS involvement. LSD therapies for systemic diseases have been developed, but efficacy does not extend to the CNS. In this study, we tested whether adeno-associated virus type 4 (AAV4) vectors could mediate global functional and pathological improvements in a murine model of...

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Identifiers

PMID: 16221840
DOI: 10.1523/JNEUROSCI.2936-05.2005
PMCID: PMC6725689

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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