Hematopoietic Stem Cell and Gene Therapy Corrects Primary Neuropathology and Behavior in Mucopolysaccharidosis IIIA Mice

Mucopolysaccharidosis IIIA (MPS IIIA or Sanfilippo disease) is a neurodegenerative disorder caused by a deficiency in the lysosomal enzyme sulfamidase (SGSH), catabolizing heparan sulfate (HS). Affected children present with severe behavioral abnormalities, sleep disturbances, and progressive neurod...

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Hlavní autoři: Langford-Smith, Alexander, Wilkinson, Fiona L, Langford-Smith, Kia J, Holley, Rebecca J, Sergijenko, Ana, Howe, Steven J, Bennett, William R, Jones, Simon A, Wraith, JE, Merry, Catherine LR, Wynn, Robert F, Bigger, Brian W
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2012
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3421066/
https://ncbi.nlm.nih.gov/pubmed/22547151
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.82
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