Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolle...
Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial
About this item
Full title
Author / Creator
Tardieu, Marc , Zérah, Michel , Gougeon, Marie-Lise , Ausseil, Jérome , de Bournonville, Stéphanie , Husson, Béatrice , Zafeiriou, Dimitrios , Parenti, Giancarlo , Bourget, Philippe , Poirier, Béatrice , Furlan, Valérie , Artaud, Cécile , Baugnon, Thomas , Roujeau, Thomas , Crystal, Ronald G , Meyer, Christian , Deiva, Kumaran and Heard, Jean-Michel
Publisher
England: Elsevier Ltd
Journal title
Language
English
Formats
Publication information
Publisher
England: Elsevier Ltd
Subjects
More information
Scope and Contents
Contents
Mucopolysaccharidosis type IIIB syndrome (also known as Sanfilippo type B syndrome) is a lysosomal storage disease resulting in progressive deterioration of cognitive acquisition after age 2–4 years. No treatment is available for the neurological manifestations of the disease. We sought to assess the safety and efficacy of a novel intracerebral gen...
Alternative Titles
Full title
Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial
Authors, Artists and Contributors
Author / Creator
Zérah, Michel
Gougeon, Marie-Lise
Ausseil, Jérome
de Bournonville, Stéphanie
Husson, Béatrice
Zafeiriou, Dimitrios
Parenti, Giancarlo
Bourget, Philippe
Poirier, Béatrice
Furlan, Valérie
Artaud, Cécile
Baugnon, Thomas
Roujeau, Thomas
Crystal, Ronald G
Meyer, Christian
Deiva, Kumaran
Heard, Jean-Michel
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_journals_1927872660
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_1927872660
Other Identifiers
ISSN
1474-4422
E-ISSN
1474-4465
DOI
10.1016/S1474-4422(17)30169-2
