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Long-term enzyme correction and lipid reduction in multiple organs of primary and secondary transplanted Fabry mice receiving transduced bone marrow cells

Fabry disease is a compelling target for gene therapy as a treatment strategy. A deficiency in the lysosomal hydrolase α-galactosidase A (α-gal A; EC 3.2.1.22) leads to impaired catabolism of α-galactosyl-terminal lipids such as globotriaosylceramide (Gb3). Patients develop vascular occlusions that...

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Udgivet i:Proc Natl Acad Sci U S A
Principais autores: Takenaka, Toshihiro, Murray, Gary J., Qin, Gangjian, Quirk, Jane M., Ohshima, Toshio, Qasba, Pankaj, Clark, Kelly, Kulkarni, Ashok B., Brady, Roscoe O., Medin, Jeffrey A.
Format: Artigo
Sprog:Inglês
Udgivet: National Academy of Sciences 2000
Fag:
Biological Sciences
Online adgang:https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC16577/
https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/10840053/
https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.120177997
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