Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector

Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector

Gaucher disease type 1 (GD1) is an inherited lysosomal disorder with multisystemic effects in patients. Hallmark symptoms include hepatosplenomegaly, cytopenias, and bone disease with varying degrees of severity. Mutations in a single gene, glucosidase beta acid 1 (GBA1), are the underlying cause fo...

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Bibliographic Details
Main Authors: Maria Dahl, Emma M.K. Smith, Sarah Warsi, Michael Rothe, Maria J. Ferraz, Johannes M.F.G. Aerts, Azadeh Golipour, Claudia Harper, Richard Pfeifer, Daniella Pizzurro, Axel Schambach, Chris Mason, Stefan Karlsson
Format: Article
Language:English
Published: Elsevier 2021-03-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S232905012030245X

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http://www.sciencedirect.com/science/article/pii/S232905012030245X

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