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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Dettagli Bibliografici
Pubblicato in:BBA Clin
Autori principali: He, Xingxuan, Dworski, Shaalee, Zhu, Changzhi, DeAngelis, Victor, Solyom, Alex, Medin, Jeffrey A., Simonaro, Calogera M., Schuchman, Edward H.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Elsevier 2017
Soggetti:
Regular Article
Accesso online: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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