Cargando…

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...

Descripción completa

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:
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