Gene therapy for metachromatic leukodystrophy
Author:
Affiliation:
1. Department of Genetic Medicine; Weill Cornell Medical College; New York New York
2. INSERM U1169; Paris France
Funder
National Institutes of Health, National Institute of Neurological Disorders and Stroke
Publisher
Wiley
Subject
Cellular and Molecular Neuroscience
Reference100 articles.
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3. The AAV vector toolkit: poised at the clinical crossroads;Asokan;Mol Ther,2012
4. Gene therapy for leukodystrophy: progress, challenges and opportunities;Aubourg;Expert Opin Orphan Drugs,2016
5. Mouse models of metachromatic leukodystrophy and adrenoleukodystrophy;Aubourg;Animal Models Dementia,2011
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