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Gene therapy: prospects for glycolipid storage diseases.

Lysosomal storage diseases comprise a group of about 40 disorders, which in most cases are due to the deficiency of a lysosomal enzyme. Since lysosomal enzymes are involved in the degradation of various compounds, the diseases can be further subdivided according to which pathway is affected. Thus, e...

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Bibliographische Detailangaben
Hauptverfasser: Gieselmann, Volkmar, Matzner, Ulrich, Klein, Diana, Mansson, Jan Eric, D'Hooge, Rudi, DeDeyn, Peter D, Lüllmann Rauch, Renate, Hartmann, Dieter, Harzer, Klaus
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2003
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC1693175/
https://ncbi.nlm.nih.gov/pubmed/12803926
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1098/rstb.2003.1277
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