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Effective cell and gene therapy in a murine model of Gaucher disease
Gaucher disease (GD) is a lysosomal storage disorder due to an inherited deficiency in the enzyme glucosylceramidase (GCase) that causes hepatosplenomegaly, cytopenias, and bone disease as key clinical symptoms. Previous mouse models with GCase deficiency have been lethal in the perinatal period or...
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| Main Authors: | , , , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
National Academy of Sciences
2006
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC1564262/ https://ncbi.nlm.nih.gov/pubmed/16954197 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0606016103 |
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