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An immune deficient mouse model for mucopolysaccharidosis IIIA (Sanfilippo syndrome)
Mucopolysaccharidosis III (MPSIII, Sanfilippo syndrome) is a devastating lysosomal storage disease that primarily affects the central nervous system. MPSIIIA is caused by loss-of-function mutations in the gene coding for sulfamidase (N-sulfoglucosamine sulfohydrolase/SGSH) resulting in SGSH enzyme d...
Autores principales: | Pollock, Kari, Noritake, Sabrina, Imai, Denise M., Pastenkos, Gabrielle, Olson, Marykate, Cary, Whitney, Yang, Sheng, Fierro, Fernando A., White, Jeannine, Graham, Justin, Dahlenburg, Heather, Johe, Karl, Nolta, Jan A. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10611714/ https://www.ncbi.nlm.nih.gov/pubmed/37891179 http://dx.doi.org/10.1038/s41598-023-45178-0 |
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