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...

Täydet tiedot

Bibliografiset tiedot

Julkaisussa:	Proc Natl Acad Sci U S A
Päätekijät:	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
Aineistotyyppi:	Artigo
Kieli:	Inglês
Julkaistu:	National Academy of Sciences 2001
Aiheet:	Biological Sciences
Linkit:	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
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