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Cell-autonomous expression of the acid hydrolase galactocerebrosidase

Lysosomal storage diseases (LSDs) are typically caused by a deficiency in a soluble acid hydrolase and are characterized by the accumulation of undegraded substrates in the lysosome. Determining the role of specific cell types in the pathogenesis of LSDs is a major challenge due to the secretion and...

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Detalhes bibliográficos
Publicado no:Proc Natl Acad Sci U S A
Main Authors: Mikulka, Christina R., Dearborn, Joshua T., Benitez, Bruno A., Strickland, Amy, Liu, Lin, Milbrandt, Jeffrey, Sands, Mark S.
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2020
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7183170/
https://ncbi.nlm.nih.gov/pubmed/32253319
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1917675117
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