Slow, continuous enzyme replacement via spinal CSF in dogs with the paediatric-onset neurodegenerative disease, MPS IIIA-Reference-Cited by-同舟云学术

Slow, continuous enzyme replacement via spinal CSF in dogs with the paediatric-onset neurodegenerative disease, MPS IIIA

Published:2016-11-10 Issue:3 Volume:40 Page:443-453
ISSN:0141-8955
Container-title:Journal of Inherited Metabolic Disease
language:en
Short-container-title:J Inherit Metab Dis

Author:

King Barbara,Marshall Neil R.,Hassiotis Sofia,Trim Paul J.,Tucker Justin,Hattersley Kathryn,Snel Marten F.,Jolly Robert D.,Hopwood John J.,Hemsley Kim M.

Funder

Shire (USA)

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Link

http://link.springer.com/content/pdf/10.1007/s10545-016-9994-1.pdf

Reference32 articles.

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3. Beard H, Luck AJ, Hassiotis S et al (2015a) Determination of the role of injection site on the efficacy of intra-CSF enzyme replacement therapy in MPS IIIA mice. Mol Genet Metab 115:33–40

4. Bhaumik M, Muller VJ, Rozaklis T et al (1999) A mouse model for mucopolysaccharidosis type III A (Sanfilippo syndrome). Glycobiology 9:1389–1396

5. Calias P, Papisov M, Pan J et al (2012) CNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: implications for neurological outcomes of lysosomal storage disorder. PLoS ONE 7:e30341

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