Improvement of biochemical and behavioral defects in the Niemann–Pick type A mouse by intraventricular infusion of MARCKS
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 brain i...
Main Authors: | Laura Trovò, Stijn Stroobants, Rudi D'Hooge, Maria Dolores Ledesma, Carlos G. Dotti |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2015-01-01
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Series: | Neurobiology of Disease |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S0969996114002800 |
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