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Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease

Sanfilippo syndrome type B (mucopolysaccharidosis type IIIB) is a recessive genetic disorder that severely affects the brain due to a deficiency in the enzyme α-N-acetylglucosaminidase (NAGLU), leading to intra-lysosomal accumulation of partially degraded heparan sulfate. There are no effective trea...

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Detalles Bibliográficos
Autores principales:	Pearse, Yewande, Clarke, Don, Kan, Shih-hsin, Le, Steven Q., Sanghez, Valentina, Luzzi, Anna, Pham, Ivy, Nih, Lina R., Cooper, Jonathan D., Dickson, Patricia I., Iacovino, Michelina
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2022
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9672419/ https://www.ncbi.nlm.nih.gov/pubmed/36419468 http://dx.doi.org/10.1016/j.omtm.2022.10.013

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