Sustained biochemical engraftment and early clinical outcomes following autologous stem cell gene therapy for mucopolysaccharidosis type IIIA

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Bibliographic Details
Published in:Molecular genetics and metabolism 2023-02, Vol.138 (2), p.107171, Article 107171
Main Authors: Jones, Simon, Kinsella, Jane, Holley, Rebecca, Potter, Jane, Booth, Claire, Buckland, Karen, Rust, Stewart, Church, Heather J., Brammeier, Kathryn L., Lee, Helena, Ford, Laura, Ellison, Stuart M., Bigger, Brian, Wynn, Robert
Format: Article
Language:English
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ISSN:1096-7192
DOI:10.1016/j.ymgme.2022.107171