Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transfer

Fabry disease is an X-linked recessive inborn metabolic disorder characterized by systemic and vascular accumulation of globotriaosylceramide (Gb(3)) caused by a deficiency of the lysosomal enzyme α-galactosidase A (α-gal A). The condition is associated with an increased morbidity and mortality due...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: Park, Jinhee, Murray, Gary J., Limaye, Advait, Quirk, Jane M., Gelderman, Monique P., Brady, Roscoe O., Qasba, Pankaj
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: The National Academy of Sciences 2003
Pynciau:
Biological Sciences
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC152313/
https://ncbi.nlm.nih.gov/pubmed/12624185
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0537900100
Tagiau: Ychwanegu Tag
Dim Tagiau, Byddwch y cyntaf i dagio'r cofnod hwn!

Eitemau Tebyg