Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy

@article{Holley2018MacrophageEA,
  title={Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy},
  author={Rebecca J. Holley and Stuart M. Ellison and Daniel Fil and C Oleary and John Henry McDermott and Nishanthi Senthivel and Alexander W W Langford-Smith and Fiona L. Wilkinson and Zelpha D'Souza and Helen Parker and Aiyin Liao and Samuel Rowlston and H{\'e}l{\`e}ne Fe Gleitz and Shih-hsin Kan and Patricia I. Dickson and Brian W Bigger},
  journal={Brain},
  year={2018},
  volume={141},
  pages={99–116}
}
  • Rebecca J. Holley, Stuart M. Ellison, +13 authors Brian W Bigger
  • Published 2018
  • Medicine
  • Brain
  • Mucopolysaccharidosis IIIB is a paediatric lysosomal storage disease caused by deficiency of the enzyme α-N-acetylglucosaminidase (NAGLU), involved in the degradation of the glycosaminoglycan heparan sulphate. Absence of NAGLU leads to accumulation of partially degraded heparan sulphate within lysosomes and the extracellular matrix, giving rise to severe CNS degeneration with progressive cognitive impairment and behavioural problems. There are no therapies. Haematopoietic stem cell transplant… CONTINUE READING

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    Current and Emerging Therapies for Mucopolysaccharidoses.

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