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Synergistic effects of treating the spinal cord and brain in CLN1 disease
Infantile neuronal ceroid lipofuscinosis (INCL, or CLN1 disease) is an inherited neurodegenerative storage disorder caused by a deficiency of the lysosomal enzyme palmitoyl protein thioesterase 1 (PPT1). It was widely believed that the pathology associated with INCL was limited to the brain, but we...
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| Publicado no: | Proc Natl Acad Sci U S A |
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| Main Authors: | , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
National Academy of Sciences
2017
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5530669/ https://ncbi.nlm.nih.gov/pubmed/28673981 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1701832114 |
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