Design of a regulated lentiviral vector for hematopoietic stem cell gene therapy of globoid cell leukodystrophy

Globoid cell leukodystrophy (GLD) is a demyelinating lysosomal storage disease due to the deficiency of the galactocerebrosidase (GALC) enzyme. The favorable outcome of hematopoietic stem and progenitor cell (HSPC)-based approaches in GLD and other similar diseases suggests HSPC gene therapy as a pr...

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Detalles Bibliográficos
Autores principales: Ungari, Silvia, Montepeloso, Annita, Morena, Francesco, Cocchiarella, Fabienne, Recchia, Alessandra, Martino, Sabata, Gentner, Bernhard, Naldini, Luigi, Biffi, Alessandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2015
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4605225/
https://www.ncbi.nlm.nih.gov/pubmed/26509184
http://dx.doi.org/10.1038/mtm.2015.38

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4605225/
https://www.ncbi.nlm.nih.gov/pubmed/26509184
http://dx.doi.org/10.1038/mtm.2015.38

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