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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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Podrobná bibliografie
Vydáno v:Ann N Y Acad Sci
Hlavní autoři: Xu, Miao, Motabar, Omid, Ferrer, Marc, Marugan, Juan J., Zheng, Wei, Ottinger, Elizabeth A.
Médium: Artigo
Jazyk:Inglês
Vydáno: 2016
Témata:
On-line přístup: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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