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EMBO Molecular Medicine|February 15, 2020
Haematopoietic stem cell gene therapy with IL-1Ra rescues cognitive loss in mucopolysaccharidosis IIIAHelen Parker, Stuart M Ellison, Rebecca J Holley, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|May 2, 2012
Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA miceAlexander Langford-Smith, Fiona L Wilkinson, Kia J Langford-Smith, et al.
Plos One|May 5, 2012
Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIBFiona L Wilkinson, Rebecca J Holley, Kia J Langford-Smith, et al.
Human Gene Therapy|April 26, 2019
An Improved Adeno-Associated Virus Vector for Neurological Correction of the Mouse Model of Mucopolysaccharidosis IIIAAnna L Gray, Claire O'Leary, Aiyin Liao, et al.
Matrix Biology : Journal of the International Society for Matrix Biology|July 12, 2019
Peptide gels of fully-defined composition and mechanics for probing cell-cell and cell-matrix interactions in vitroJ C Ashworth, J L Thompson, J R James, et al.
Brain : a Journal of Neurology|November 30, 2017
Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapyRebecca J Holley, Stuart M Ellison, Daniel Fil, et al.
The Journal of Clinical Investigation|June 20, 2025
Neuraminidase 1 secondary deficiency contributes to CNS pathology in neurological mucopolysaccharidoses via brain protein hypersialylationTianMeng Xu, Rachel Heon-Roberts, Travis Moore, et al.
ESMO Real World Data and Digital Oncology|February 6, 2026
Challenges and opportunities for real-world evidence in clinical oncology-a view from the UK: proceedings of a national workshopM Craddock, C Dempsey, D Abdulwahid, et al.
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