Macrophages Expressing GALC Improve Peripheral Krabbe Disease by a Mechanism Independent of Cross-Correction

Many therapies for lysosomal storage disorders rely on cross-correction of lysosomal enzymes. In globoid cell leukodystrophy (GLD), mutations in GALC cause psychosine accumulation, inducing demyelination, a neuroinflammatory “globoid” reaction and neurodegeneration. The efficiency of GALC cross-corr...

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Publicado no:Neuron
Main Authors: Weinstock, Nadav I., Shin, Daesung, Dhimal, Narayan, Hong, Xinying, Irons, Eric E., Silvestri, Nicholas J., Reed, Chelsey B., Nguyen, Duc, Sampson, Oliver, Cheng, Yung-Chih, Lau, Joseph T.Y., Bongarzone, Ernesto R., Kofler, Julia, Escolar, Maria L., Gelb, Michael H., Wrabetz, Lawrence, Feltri, M. Laura
Formato: Artigo
Idioma:Inglês
Publicado em: 2020
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7924901/
https://ncbi.nlm.nih.gov/pubmed/32375064
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.neuron.2020.03.031
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