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Gene Therapy Approaches for Lysosomal Storage Disease: Next-Generation Treatment
Lysosomal storage diseases are a group of rare inborn errors of metabolism resulting from deficiency in normal lysosomal function. These diseases are characterized by progressive accumulation of storage material within the lysosomes of affected cells, ultimately leading to cellular dysfunction. Mult...
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| Main Authors: | , , , |
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| פורמט: | Artigo |
| שפה: | Inglês |
| יצא לאור: |
Mary Ann Liebert, Inc.
2012
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| נושאים: | |
| גישה מקוונת: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3413894/ https://ncbi.nlm.nih.gov/pubmed/22794786 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2012.140 |
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