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604. Intrathecal AAV9-Mediated Gene Delivery Corrects Lysosomal Storage Throughout the CNS in a Large Animal Model of Mucopolysaccharidosis Type I
Molecular Therapy
- United States
doi 10.1016/s1525-0016(16)35617-9
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Abstract
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Categories
Molecular Medicine
Molecular Biology
Pharmacology
Medicine
Genetics
Drug Discovery
Date
May 1, 2014
Authors
Unknown
Publisher
Elsevier BV