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Neural stem cells for disease modeling of Wolman disease and evaluation of therapeutics
BACKGROUND: Wolman disease (WD) is a rare lysosomal storage disorder that is caused by mutations in the LIPA gene encoding lysosomal acid lipase (LAL). Deficiency in LAL function causes accumulation of cholesteryl esters and triglycerides in lysosomes. Fatality usually occurs within the first year o...
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| Опубликовано в: : | Orphanet J Rare Dis |
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| Главные авторы: | , , , , , , , |
| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
BioMed Central
2017
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| Предметы: | |
| Online-ссылка: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5490176/ https://ncbi.nlm.nih.gov/pubmed/28659158 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-017-0670-9 |
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