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

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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
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container_title Molecular genetics and metabolism
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creator 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
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doi_str_mv 10.1016/j.ymgme.2022.107171
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title Sustained biochemical engraftment and early clinical outcomes following autologous stem cell gene therapy for mucopolysaccharidosis type IIIA
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