Targeting Wolman Disease and Cholesteryl Ester Storage Disease: Disease Pathogenesis and Therapeutic Development

Wolman disease (WD) and cholesteryl ester storage disease (CESD) are lysosomal storage diseases (LSDs) caused by a deficiency in lysosomal acid lipase (LAL) due to mutations in the LIPA gene. This enzyme is critical to the proper degradation of cholesterol in the lysosome. LAL function is completely...

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Detalles Bibliográficos
Autores principales: Aguisanda, Francis, Thorne, Natasha, Zheng, Wei
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Bentham Open 2017
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5362971/
https://www.ncbi.nlm.nih.gov/pubmed/28401034
http://dx.doi.org/10.2174/2213988501711010001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5362971/
https://www.ncbi.nlm.nih.gov/pubmed/28401034
http://dx.doi.org/10.2174/2213988501711010001

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