Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy

Globoid cell leukodystrophy (GLD) is an inherited lysosomal storage disease caused by β-galactocerebrosidase (GALC) deficiency. Gene therapy (GT) should provide rapid, extensive and lifetime GALC supply in central nervous system (CNS) tissues to prevent or halt irreversible neurologic progression. H...

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Huvudupphovsmän: Lattanzi, Annalisa, Salvagno, Camilla, Maderna, Claudio, Benedicenti, Fabrizio, Morena, Francesco, Kulik, Willem, Naldini, Luigi, Montini, Eugenio, Martino, Sabata, Gritti, Angela
Materialtyp: Artigo
Språk:Inglês
Publicerad: Oxford University Press 2014
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Articles
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC4030779/
https://ncbi.nlm.nih.gov/pubmed/24463623
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddu034
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