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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Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: BioMed Central 2012
Soggetti:
Research
Accesso online: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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