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Gene Therapy for Neurologic Manifestations of Mucopolysaccharidoses
INTRODUCTION: Mucopolysaccharidoses are a family of lysosomal disorders caused by mutations in genes that encode enzymes involved in the catabolism of glycoaminoglycans. These mutations affect multiple organ systems and can be particularly deleterious to the nervous system. At the present time, enzy...
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| 發表在: | Expert Opin Drug Deliv |
|---|---|
| Main Authors: | , , , , , |
| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
2014
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| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4393078/ https://ncbi.nlm.nih.gov/pubmed/25510418 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1517/17425247.2015.966682 |
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