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Journal of Inherited Metabolic Disease
Published in: Journal of Inherited Metabolic Disease 6/2016

01-11-2016 | Highlights

News and views

Author: Peter Witters

Published in: Journal of Inherited Metabolic Disease | Issue 6/2016

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Excerpt

In The Lancet the interim analysis of a promising trial of lentiviral hematopoietic stem cell gene therapy in early-onset metachromatic leukodystrophy (MLD) shows impressive results (Sessa et al 2016). Its innovative approach opens the path to therapies for lysosomal storage disorder with central nervous system (CNS) involvement. …
Literature
Cartier N, Hacein-Bey-Abina S, Bartholomae CC et al (2009) Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 326:818–823CrossRefPubMed
Krageloh-Mann I, Groeschel S (2016) Therapies of lysosomal storage disorders targeting the brain. Lancet 388:440–442CrossRefPubMed
Sessa M, Lorioli L, Fumagalli F et al (2016) Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial. Lancet 388:476–487CrossRefPubMed
Metadata
Title
News and views
Author
Peter Witters
Publication date
01-11-2016
Publisher
Springer Netherlands
Published in
Journal of Inherited Metabolic Disease / Issue 6/2016
Print ISSN: 0141-8955
Electronic ISSN: 1573-2665
DOI
https://doi.org/10.1007/s10545-016-9983-4

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