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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Detalhes bibliográficos
Publicado no:BBA Clin
Main Authors: He, Xingxuan, Dworski, Shaalee, Zhu, Changzhi, DeAngelis, Victor, Solyom, Alex, Medin, Jeffrey A., Simonaro, Calogera M., Schuchman, Edward H.
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2017
Assuntos:
Regular Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5338723/
https://ncbi.nlm.nih.gov/pubmed/28275553
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.bbacli.2017.02.001
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