Enzyme replacement therapy for Farber disease: Proof-of-concept studies in cells and mice

A series of studies were carried out in Farber disease (OMIM #228000) cells and mice to evaluate the feasibility of enzyme replacement therapy (ERT) for this disorder. Media from Chinese hamster ovary (CHO) cells overexpressing human recombinant acid ceramidase (rhAC) was used to treat fibroblasts f...

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Detalles Bibliográficos
Autores principales: He, Xingxuan, Dworski, Shaalee, Zhu, Changzhi, DeAngelis, Victor, Solyom, Alex, Medin, Jeffrey A., Simonaro, Calogera M., Schuchman, Edward H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2017
Materias:
Regular Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5338723/
https://www.ncbi.nlm.nih.gov/pubmed/28275553
http://dx.doi.org/10.1016/j.bbacli.2017.02.001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5338723/
https://www.ncbi.nlm.nih.gov/pubmed/28275553
http://dx.doi.org/10.1016/j.bbacli.2017.02.001

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