Lentivector Transduction Improves Outcomes Over Transplantation of Human HSCs Alone in NOD/SCID/Fabry Mice

Fabry disease is a lysosomal storage disorder caused by a deficiency of α-galactosidase A (α-gal A) activity that results in progressive globotriaosylceramide (Gb(3)) deposition. We created a fully congenic nonobese diabetic (NOD)/severe combined immunodeficiency (SCID)/Fabry murine line to facilita...

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Autori principali: Pacienza, Natalia, Yoshimitsu, Makoto, Mizue, Nobuo, Au, Bryan CY, Wang, James CM, Fan, Xin, Takenaka, Toshihiro, Medin, Jeffrey A
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2012
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3393855/
https://ncbi.nlm.nih.gov/pubmed/22472949
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.64
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