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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Detalhes bibliográficos
Publicado no:EMBO Mol Med
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2020
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Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7059006/
https://ncbi.nlm.nih.gov/pubmed/32057196
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.201911185
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