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Biochemical Engraftment and Clinical Outcomes Following Ex-Vivo Autologous Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIIA

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Bibliographic Details
Published in:Blood 2022-11, Vol.140 (Supplement 1), p.1897-1898
Main Authors: Wynn, Robert F, Kinsella, Jane L, Holley, Rebecca J, Potter, Jane, Thrasher, Adrian J., Booth, Claire, Buckland, Karen, Izotova, Natalia, Rust, Stewart, Weisberg, Daniel, Church, Heather J, Tylee, Karen L, Brammeier, Kathryn L, Lee, Helena, Ford, Laura, Sadegholnejat, Atusa, Jones, Simon A, Ellison, Stuart, Bigger, Brian
Format: Article
Language:English
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ISSN:0006-4971
1528-0020
DOI:10.1182/blood-2022-168672