Intravitreal Gene Therapy Reduces Lysosomal Storage in Specific Areas of the CNS in Mucopolysaccharidosis VII Mice

The mucopolysaccharidoses (MPSs) are lysosomal storage diseases resulting from impaired catabolism of sulfated glycosaminoglycans. MPS VII mice lack lysosomal β-glucuronidase (GUSB) activity, leading to the accumulation of partially degraded chondroitin, dermatan, and heparan sulfates in most tissue...

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Foilsithe in:J Neurosci
Main Authors: Hennig, Anne K., Levy, Beth, Ogilvie, Judith Mosinger, Vogler, Carole A., Galvin, Nancy, Bassnett, Steven, Sands, Mark S.
Formáid: Artigo
Teanga:Inglês
Foilsithe: Society for Neuroscience 2003
Ábhair:
ARTICLE
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC6742327/
https://ncbi.nlm.nih.gov/pubmed/12716937
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1523/JNEUROSCI.23-08-03302.2003
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