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Musings on genome medicine: enzyme-replacement therapy of the lysosomal storage diseases
The lysosomal storage diseases, such as Gaucher's disease, mucopolysaccharidosis I, II and IV, Fabry's disease, and Pompe's disease, are rare inherited disorders whose symptoms result from enzyme deficiency causing lysosomal accumulation. Until effective gene-replacement therapy is de...
| Autores principales: | , |
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| Formato: | Texto |
| Lenguaje: | English |
| Publicado: |
BioMed Central
2009
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2808730/ https://www.ncbi.nlm.nih.gov/pubmed/20017892 http://dx.doi.org/10.1186/gm114 |