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Authors
Mandolfo, Oriana https://orcid.org/0000-0003-3726-9911
Parker, Helen
Aguado, Èlia
Ishikawa Learmonth, Yuko https://orcid.org/0009-0004-1016-5324
Liao, Ai Yin
O’Leary, Claire
Ellison, Stuart
Forte, Gabriella https://orcid.org/0000-0002-6656-6328
Taylor, Jessica
Wood, Shaun
Searle, Rachel
Holley, Rebecca J https://orcid.org/0000-0003-4150-3299
Boutin, Hervé
Bigger, Brian W https://orcid.org/0000-0002-9708-1112
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Funding
Funding for this research was provided by:
Penn | Penn Med | Orphan Disease Center, Perelman School of Medicine, University of Pennsylvania
Neuroscience Research Institute, Manchester
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Article History
Received: 7 July 2023
Revised: 30 May 2024
Accepted: 4 June 2024
First Online: 18 June 2024
Disclosure and competing interests statement
: BWB is a recipient of licence fees, and unrestricted clinical trial grants from Orchard Therapeutics and AVROBIO for haematopoietic stem cell gene therapies in MPS IIIA and MPSII, respectively, unrelated to this paper. The authors declare no competing interests.
