Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice

Sanfilippo syndrome type B (mucopolysaccharidosis type IIIB [MPS IIIB]) is a lysosomal storage disorder primarily affecting the brain that is caused by a deficiency in the enzyme α-N-acetylglucosaminidase (NAGLU), leading to intralysosomal accumulation of heparan sulfate. There are currently no trea...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Clarke, Don, Pearse, Yewande, Kan, Shih-hsin, Le, Steven Q., Sanghez, Valentina, Cooper, Jonathan D., Dickson, Patricia I., Iacovino, Michelina
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6076361/
https://ncbi.nlm.nih.gov/pubmed/30101150
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.06.005
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