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

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

Abstract 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 accu...

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Main Authors: Helen Parker, Stuart M Ellison, Rebecca J Holley, Claire O'Leary, Aiyin Liao, Jalal Asadi, Emily Glover, Arunabha Ghosh, Simon Jones, Fiona L Wilkinson, David Brough, Emmanuel Pinteaux, Hervé Boutin, Brian W Bigger
Format: Article
Language:English
Published: Springer Nature 2020-02-01
Series:EMBO Molecular Medicine
Subjects:
cognitive decline
haematopoietic stem cell gene therapy
inflammasome
interleukin‐1 receptor antagonist
mucopolysaccharidosis
Online Access:https://doi.org/10.15252/emmm.201911185
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https://doi.org/10.15252/emmm.201911185

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