Validation of an assay to measure iduronate-2-sulfatase activity in cerebrospinal fluid to assess the efficacy of a hematopoietic stem cell (HSC) gene therapy

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Bibliographic Details
Published in:Molecular genetics and metabolism 2023-02, Vol.138 (2), p.107106, Article 107106
Main Authors: French, Stephanie A., Brammeier, Kathryn L., Jones, Simon, Tylee, Karen L., Church, Heather J.
Format: Article
Language:English
Online Access:Get full text
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ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2022.107106