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...

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Detalles Bibliográficos
Autores principales: Nathan, David G, Orkin, Stuart H
Formato: Texto
Lenguaje:English
Publicado: BioMed Central 2009
Materias:
Musings
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

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2808730/
https://www.ncbi.nlm.nih.gov/pubmed/20017892
http://dx.doi.org/10.1186/gm114

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