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CNS-directed gene therapy for lysosomal storage diseases
Lysosomal storage diseases (LSDs) are a group of inherited metabolic disorders usually caused by deficient activity of a single lysosomal enzyme. As most lysosomal enzymes are ubiquitously expressed, a deficiency in a single enzyme can affect multiple organ systems, including the central nervous sys...
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| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
2008
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3340572/ https://ncbi.nlm.nih.gov/pubmed/18339183 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/j.1651-2227.2008.00660.x |
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