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Authors
Milani, Michela https://orcid.org/0000-0002-0363-678X
Fabiano, Anna https://orcid.org/0000-0002-7604-6967
Perez-Rodriguez, Marta https://orcid.org/0009-0007-8115-4093
Hernandez, Raisa Jofra
Zecchillo, Alessandra
Zonari, Erika
Ottonello, Sofia
Basso-Ricci, Luca
Canepari, Cesare
Volpin, Monica
Iannello, Valeria
Capo, Valentina https://orcid.org/0000-0002-1851-1429
Quaranta, Pamela
Seffin, Luca
Russo, Fabio https://orcid.org/0000-0001-5280-5677
Biffi, Mauro https://orcid.org/0000-0002-6914-2854
Ormoli, Leonardo
Brombin, Chiara
Carlucci, Filippo
Forlino, Antonella
Filibian, Marta
Montini, Eugenio https://orcid.org/0000-0003-1771-6067
Scala, Serena
Villa, Anna
Bueren, Juan Antonio https://orcid.org/0000-0002-3228-7013
Rio, Paula https://orcid.org/0000-0002-5424-5543
Aiuti, Alessandro https://orcid.org/0000-0002-5398-1717
Cantore, Alessio https://orcid.org/0000-0002-9741-997X
Naldini, Luigi https://orcid.org/0000-0002-7835-527X
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Article History
Received: 8 February 2024
Accepted: 24 April 2025
First Online: 28 May 2025
Competing interests
: P.R. and J.A.B. receive funding and have licensed the PGK.FANCA-LV to Rocket Pharmaceuticals and are inventors on patents filed by CIEMAT, CIBERER and Fundación Jiménez Díaz, and may be entitled to receive financial benefits from the licensing of such patents. J.A.B. is consultant of Rocket Pharmaceuticals, A.A. is the principal investigator of clinical trials with LV-transduced haematopoietic stem cells sponsored by Orchard Therapeutics or Fondazione Telethon. L.N. and A.C. are inventors on patent applications submitted by Fondazione Telethon or the San Raffaele Scientific Institute on LV technology for in vivo use related to the work presented in this paper. The other authors declare no competing interests.
