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Disease models for the development of therapies for lysosomal storage diseases

Lysosomal storage diseases (LSDs) are a group of rare diseases in which the function of the lysosome is disrupted by the accumulation of macromolecules. The complexity underlying the pathogenesis of LSDs and the small, often pediatric, population of patients make the development of therapies for the...

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Detalhes bibliográficos
Publicado no:Ann N Y Acad Sci
Main Authors: Xu, Miao, Motabar, Omid, Ferrer, Marc, Marugan, Juan J., Zheng, Wei, Ottinger, Elizabeth A.
Formato: Artigo
Idioma:Inglês
Publicado em: 2016
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5287412/
https://ncbi.nlm.nih.gov/pubmed/27144735
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/nyas.13052
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