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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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| Publicado en: | Mol Genet Metab |
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| Autores principales: | , |
| Formato: | Artigo |
| Lenguaje: | Inglês |
| Publicado: |
2014
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| Materias: | |
| Acceso en línea: | 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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