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Treatment of adult MPSI mouse brains with IDUA-expressing mesenchymal stem cells decreases GAG deposition and improves exploratory behavior

BACKGROUND: Mucopolysaccharidosis type I (MPSI) is caused by a deficiency in alpha-L iduronidase (IDUA), which leads to lysosomal accumulation of the glycosaminoglycans (GAGs) dermatan and heparan sulfate. While the currently available therapies have good systemic effects, they only minimally affect...

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Библиографические подробности
Главные авторы: da Silva, Flávia Helena, Pereira, Vanessa Gonçalves, Yasumura, Eduardo G, Tenório, Lígia Zacchi, de Carvalho, Leonardo Pinto, Lisboa, Bianca Cristina Garcia, Matsumoto, Priscila Keiko, Stilhano, Roberta Sessa, Samoto, Vivian Y, Calegare, Bruno Frederico Aguilar, Brandão, Letícia de Campos, D’Almeida, Vânia, Filippo, Thaís RM, Porcionatto, Marimélia, Toma, Leny, Nader, Helena Bonciani, Valero, Valderez Bastos, Camassola, Melissa, Nardi, Nance Beyer, Han, Sang Won
Формат: Artigo
Язык:Inglês
Опубликовано: BioMed Central 2012
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Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC3404940/
https://ncbi.nlm.nih.gov/pubmed/22520214
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1479-0556-10-2
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