Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice

Fabry disease is a lysosomal storage disorder caused by a deficiency of the lysosomal enzyme α-galactosidase A (α-gal A). This enzyme deficiency leads to impaired catabolism of α-galactosyl-terminal lipids such as globotriaosylceramide (Gb3). Patients develop painful neuropathy and vascular occlusio...

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Autori principali: Jung, Sung-Chul, Han, Ina P., Limaye, Advait, Xu, Ruian, Gelderman, Monique P., Zerfas, Patricia, Tirumalai, Kamala, Murray, Gary J., During, Matthew J., Brady, Roscoe O., Qasba, Pankaj
Natura: Artigo
Lingua:Inglês
Pubblicazione: The National Academy of Sciences 2001
Soggetti:
Biological Sciences
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC30197/
https://ncbi.nlm.nih.gov/pubmed/11226298
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.051634498
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