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Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier
Most lysosomal storage disorders affect the nervous system as well as other tissues and organs of the body. Previously, the complexities of these diseases, particularly in treating neurologic abnormalities, were too great to surmount. However, based on recent developments there are realistic expecta...
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| Published in: | Mol Genet Metab |
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| Main Authors: | , |
| Format: | Artigo |
| Language: | Inglês |
| Published: |
2014
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| Subjects: | |
| Online Access: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4312729/ https://ncbi.nlm.nih.gov/pubmed/25410058 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2014.09.011 |
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