Myeloid/Microglial Driven Autologous Hematopoietic Stem Cell Gene Therapy Corrects a Neuronopathic Lysosomal Disease

Mucopolysaccharidosis type IIIA (MPSIIIA) is a lysosomal storage disorder caused by mutations in N-sulfoglucosamine sulfohydrolase (SGSH), resulting in heparan sulfate (HS) accumulation and progressive neurodegeneration. There are no treatments. We previously demonstrated improved neuropathology in...

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Bibliographische Detailangaben
Hauptverfasser: Sergijenko, Ana, Langford-Smith, Alexander, Liao, Ai Y, Pickford, Claire E, McDermott, John, Nowinski, Gabriel, Langford-Smith, Kia J, Merry, Catherine LR, Jones, Simon A, Wraith, J Edmond, Wynn, Robert F, Wilkinson, Fiona L, Bigger, Brian W
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2013
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3808137/
https://ncbi.nlm.nih.gov/pubmed/23748415
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.141
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