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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Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: The National Academy of Sciences 2000
Assuntos:
Biological Sciences
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC16577/
https://ncbi.nlm.nih.gov/pubmed/10840053
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