Acknowledgements
P.
This work was supported by Fondazione Telethon (TIGET Core Grant B2, A.A.), the Italian Ministero della Salute (Programma di Rete, NET-2011-02350069, A.A.), the European Commission (ERARE-3-JTC 2015 EUROCID, A.A.) and Else Kröner-Fresenius-Stiftung (EKFS) prize (A.A.). We thank F. Ciceri, ME Bernardo and all medical and nursing staff of the Pediatric Immunohematology and Bone Marrow Transplantation Unit of the San Raffaele Scientific Institute and the San Raffaele Stem Cell Programme; S. Zancan, M. Casiraghi, G. Tomaselli and all Sr-TIGET Clinical Trial Office personnel for clinical trial management and support; the OTL team for revising the manuscript; M. Gabaldo and G. Farinelli for support with project management; and P. Massariello, G. Vallanti, M. Manfredini and other MolMed staff for patient cell manipulation. We thank C. Waskow for her advice in handling NSGW41 mice. We thank C. Villa, E. Canonico and S. Di Terlizzi of the Flow Cytometry Resource, Advanced Cytometry Technical Applications Laboratory (FRACTAL) at Ospedale San Raffaele for cell sorting and technical help with instrumentation. We are indebted to the patients and their families for their commitment.