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

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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Bibliographic Details
Main Authors: Silvia Ungari, Annita Montepeloso, Francesco Morena, Fabienne Cocchiarella, Alessandra Recchia, Sabata Martino, Bernhard Gentner, Luigi Naldini, Alessandra Biffi
Format: Article
Language:English
Published: Elsevier 2015-01-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S232905011630050X

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http://www.sciencedirect.com/science/article/pii/S232905011630050X

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