Received: 7 October 2020
Accepted: 9 March 2021
First Online: 19 March 2021
: Written informed consent following local medical ethics committee guidelines was obtained for all patients.
: Parents of the patients gave their consent for publication.
: Two co-authors have conflicts of interests however, not related to this article. JJB reports personal fees from Avrobio, Bluerock, Takeda, Magenta, Bluebird Bio, Advanced Clinical and from Omeros, all outside the submitted work. WT reports personal fees from Pfizer, GSK, Roche Diagnostics/Ventana Merck Sharp Dohme, Novartis, Lilly Oncology, Boehringer Ingelheim, Astra-Zeneca, Bristol-Myers-Squibb, and from AbbVie, all outside the submitted work. None of the other authors have any conflits of interest.