Combined gene/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy

Globoid cell leukodystrophy (GLD) is a lysosomal storage disease caused by deficient activity of β-galactocerebrosidase (GALC). The infantile forms manifest with rapid and progressive central and peripheral demyelination, which represent a major hurdle for any treatment approach. We demonstrate here...

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Vydáno v:Hum Mol Genet
Hlavní autoři: Ricca, Alessandra, Rufo, Nicole, Ungari, Silvia, Morena, Francesco, Martino, Sabata, Kulik, Wilem, Alberizzi, Valeria, Bolino, Alessandra, Bianchi, Francesca, Del Carro, Ubaldo, Biffi, Alessandra, Gritti, Angela
Médium: Artigo
Jazyk:Inglês
Vydáno: Oxford University Press 2015
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4498152/
https://ncbi.nlm.nih.gov/pubmed/25749991
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddv086
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