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Haematopoietic stem cell gene therapy with IL‐1Ra rescues cognitive loss in mucopolysaccharidosis IIIA

Mucopolysaccharidosis IIIA is a neuronopathic lysosomal storage disease, characterised by heparan sulphate and other substrates accumulating in the brain. Patients develop behavioural disturbances and cognitive decline, a possible consequence of neuroinflammation and abnormal substrate accumulation....

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Detalles Bibliográficos
Autores principales:	Parker, Helen, Ellison, Stuart M, Holley, Rebecca J, O'Leary, Claire, Liao, Aiyin, Asadi, Jalal, Glover, Emily, Ghosh, Arunabha, Jones, Simon, Wilkinson, Fiona L, Brough, David, Pinteaux, Emmanuel, Boutin, Hervé, Bigger, Brian W
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2020
Materias:	Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7059006/ https://www.ncbi.nlm.nih.gov/pubmed/32057196 http://dx.doi.org/10.15252/emmm.201911185

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