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Authors
Budinger, Dimitri https://orcid.org/0000-0001-7002-1091
Puigdevall, Pau https://orcid.org/0000-0002-8687-4942
Hall, George T. https://orcid.org/0000-0002-4828-0668
Roth, Charlotte https://orcid.org/0000-0003-0227-3339
Xenakis, Theodoros https://orcid.org/0000-0003-3682-8873
Marrosu, Elena
Jerber, Julie
Di Domenico, Alessandro
Picco, Francesca
Kilpinen, Helena https://orcid.org/0000-0001-6692-6154
Castellano, Sergi
Kurian, Manju A. https://orcid.org/0000-0003-3529-5075
Barral, Serena https://orcid.org/0000-0002-1430-3614
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Funding
Funding for this research was provided by:
DH | National Institute for Health Research
Sir Jules Thorn Charitable Trust
Rosetrees Trust
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Article History
Received: 8 November 2024
Accepted: 5 December 2025
First Online: 3 February 2026
Competing interests
: M.A.K. is a founder of and consultant to Bloomsbury Genetic Therapies. She has received honoraria from PTC for sponsored symposia and provided consultancy. The remaining authors declare no competing interests.
