Is the eye a window to the brain in Sanfilippo syndrome?

Is the eye a window to the brain in Sanfilippo syndrome?

Abstract 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 n...

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Bibliographic Details
Main Authors: Helen Beard, Glyn Chidlow, Daniel Neumann, Nazzmer Nazri, Meghan Douglass, Paul J. Trim, Marten F. Snel, Robert J. Casson, Kim M. Hemsley
Format: Article
Language:English
Published: BMC 2020-11-01
Series:Acta Neuropathologica Communications
Subjects:
Retina
Mucopolysaccharidosis type III
Central nervous system
Eye
Lysosomal
Online Access:http://link.springer.com/article/10.1186/s40478-020-01070-w

Internet

http://link.springer.com/article/10.1186/s40478-020-01070-w

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