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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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Detalles Bibliográficos
Autores principales:	Beard, Helen, Chidlow, Glyn, Neumann, Daniel, Nazri, Nazzmer, Douglass, Meghan, Trim, Paul J., Snel, Marten F., Casson, Robert J., Hemsley, Kim M.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2020
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7672954/ https://www.ncbi.nlm.nih.gov/pubmed/33203474 http://dx.doi.org/10.1186/s40478-020-01070-w

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