Preselective gene therapy for Fabry disease

Fabry disease is a lipid storage disorder resulting from mutations in the gene encoding the enzyme α-galactosidase A (α-gal A; EC 3.2.1.22). We previously have demonstrated long-term α-gal A enzyme correction and lipid reduction mediated by therapeutic ex vivo transduction and transplantation of hem...

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Hlavní autoři: Qin, Gangjian, Takenaka, Toshihiro, Telsch, Kimberly, Kelley, Leslie, Howard, Tazuko, Levade, Thierry, Deans, Robert, Howard, Bruce H., Malech, Harry L., Brady, Roscoe O., Medin, Jeffrey A.
Médium: Artigo
Jazyk:Inglês
Vydáno: The National Academy of Sciences 2001
Témata:
Biological Sciences
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC30670/
https://ncbi.nlm.nih.gov/pubmed/11248095
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.061020598
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