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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...

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Détails bibliographiques
Auteurs principaux: Park, Jinhee, Murray, Gary J., Limaye, Advait, Quirk, Jane M., Gelderman, Monique P., Brady, Roscoe O., Qasba, Pankaj
Format: Artigo
Langue:Inglês
Publié: The National Academy of Sciences 2003
Sujets:
Accès en ligne: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
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