Flávia Helena da Silva, Vanessa Gonçalves Pereira, Eduardo G Yasumura, Lígia Zacchi Tenório, Leonardo Pinto de Carvalho, Bianca Cristina Garcia Lisboa, Priscila Keiko Matsumoto, Roberta Sessa Stilhano, Vivian Y Samoto, Bruno Frederico Aguilar Calegare, Letícia de Campos Brandão, Vânia D'Almeida, Thaís Rm Filippo, Marimélia Porcionatto, Leny Toma, Helena Bonciani Nader, Valderez Bastos Valero, Melissa Camassola, Nance Beyer Nardi, Sang Won Han
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 the neurodegenerative process. Based on the neuroprotective and tissue regenerative properties of mesenchymal stem cells (MSCs), we hypothesized that the administration of MSCs transduced with a murine leukemia virus (MLV) vector expressing IDUA to IDUA KO mouse brains could reduce GAG deposition in the brain and, as a result, improve neurofunctionality, as measured by exploratory activity...
2012: Genetic Vaccines and Therapy