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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Main Authors: Neri, Margherita, Ricca, Alessandra, di Girolamo, Ilaria, Alcala'-Franco, Beatriz, Cavazzin, Chiara, Orlacchio, Aldo, Martino, Sabata, Naldini, Luigi, Gritti, Angela
Formáid: Artigo
Teanga:Inglês
Foilsithe: Wiley Subscription Services, Inc., A Wiley Company 2011
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Original Research: Regenerative Medicine
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC3229988/
https://ncbi.nlm.nih.gov/pubmed/21809420
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/stem.701
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