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