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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Detalles Bibliográficos
Autores principales: Clarke, Don, Pearse, Yewande, Kan, Shih-hsin, Le, Steven Q., Sanghez, Valentina, Cooper, Jonathan D., Dickson, Patricia I., Iacovino, Michelina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6076361/
https://www.ncbi.nlm.nih.gov/pubmed/30101150
http://dx.doi.org/10.1016/j.omtm.2018.06.005

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6076361/
https://www.ncbi.nlm.nih.gov/pubmed/30101150
http://dx.doi.org/10.1016/j.omtm.2018.06.005

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