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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Detalhes bibliográficos
Publicado no:J Neurosci
Main Authors: Hennig, Anne K., Levy, Beth, Ogilvie, Judith Mosinger, Vogler, Carole A., Galvin, Nancy, Bassnett, Steven, Sands, Mark S.
Formato: Artigo
Idioma:Inglês
Publicado em: Society for Neuroscience 2003
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ARTICLE
Acesso em linha: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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