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Is the eye a window to the brain in Sanfilippo syndrome?
Sanfilippo syndrome is an untreatable form of childhood-onset dementia. Whilst several therapeutic strategies are being evaluated in human clinical trials including i.v. delivery of AAV9-based gene therapy, an urgent unmet need is the availability of non-invasive, quantitative measures of neurodegen...
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| 出版年: | Acta Neuropathol Commun |
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| 主要な著者: | , , , , , , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
BioMed Central
2020
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7672954/ https://ncbi.nlm.nih.gov/pubmed/33203474 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s40478-020-01070-w |
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