Lentivector Iterations and Pre-Clinical Scale-Up/Toxicity Testing: Targeting Mobilized CD34(+) Cells for Correction of Fabry Disease

Fabry disease is a rare lysosomal storage disorder (LSD). We designed multiple recombinant lentivirus vectors (LVs) and tested their ability to engineer expression of human α-galactosidase A (α-gal A) in transduced Fabry patient CD34(+) hematopoietic cells. We further investigated the safety and eff...

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Pubblicato in:Mol Ther Methods Clin Dev
Autori principali: Huang, Ju, Khan, Aneal, Au, Bryan C., Barber, Dwayne L., López-Vásquez, Lucía, Prokopishyn, Nicole L., Boutin, Michel, Rothe, Michael, Rip, Jack W., Abaoui, Mona, Nagree, Murtaza S., Dworski, Shaalee, Schambach, Axel, Keating, Armand, West, Michael L., Klassen, John, Turner, Patricia V., Sirrs, Sandra, Rupar, C. Anthony, Auray-Blais, Christiane, Foley, Ronan, Medin, Jeffrey A.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2017
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5453867/
https://ncbi.nlm.nih.gov/pubmed/28603745
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2017.05.003
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