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Article

Improvement of biochemical and behavioral defects in the Niemann-Pick type A mouse by intraventricular infusion of MARCKS.

Neurobiology of disease - 1 Jan 2015

Trovò Laura, Stroobants Stijn, D'Hooge Rudi, Ledesma Maria Dolores, Dotti Carlos G

Abstract excerpt

Niemann-Pick disease type A (NPDA) is a fatal disease due to mutations in the acid sphingomyelinase (ASM) gene, which triggers the abnormal accumulation of sphingomyelin (SM) in lysosomes and the plasma membrane of mutant cells. Although the disease affects multiple organs, the impact on the brai...

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Source

Identifiers

PMID: 25251606
DOI: 10.1016/j.nbd.2014.09.008

Genes

MARCKS

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026. Prepared record publication-25251606-2026-09-04.2. Manifest publication-graph-cohort-2026-09-04.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 4 Sept 2026.

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