QR-kod

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...

Full beskrivning

Sparad:
I publikationen:Proc Natl Acad Sci U S A
Huvudupphov: 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
Materialtyp: Artigo
Språk:Inglês
Utgiven: National Academy of Sciences 2001
Ämnen:
Biological Sciences
Länkar:https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC30197/
https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/11226298/
https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.051634498
Taggar: Lägg till en tagg
Inga taggar, Lägg till första taggen!

Liknande verk