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Neural Stem Cell Gene Therapy Ameliorates Pathology and Function in a Mouse Model of Globoid Cell Leukodystrophy

Murine neural stem cells (mNSCs), either naive or genetically modified to express supranormal levels of β-galactocerebrosidase (GALC), were transplanted into the brain of Twitcher mice, a murine model of globoid cell leukodystrophy, a severe sphingolipidosis. Cells engrafted long-term into the host...

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Detalles Bibliográficos
Autores principales:	Neri, Margherita, Ricca, Alessandra, di Girolamo, Ilaria, Alcala'-Franco, Beatriz, Cavazzin, Chiara, Orlacchio, Aldo, Martino, Sabata, Naldini, Luigi, Gritti, Angela
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Wiley Subscription Services, Inc., A Wiley Company 2011
Materias:	Original Research: Regenerative Medicine
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3229988/ https://www.ncbi.nlm.nih.gov/pubmed/21809420 http://dx.doi.org/10.1002/stem.701

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