Pontoizeau, Clément
Simon-Sola, Marcelo
Gaborit, Clovis
Nguyen, Vincent https://orcid.org/0000-0002-2084-5436
Rotaru, Irina
Tual, Nolan
Colella, Pasqualina https://orcid.org/0000-0002-4268-2391
Girard, Muriel
Biferi, Maria-Grazia
Arnoux, Jean-Baptiste
Rötig, Agnès https://orcid.org/0000-0003-0589-0703
Ottolenghi, Chris
de Lonlay, Pascale
Mingozzi, Federico
Cavazzana, Marina https://orcid.org/0000-0002-0264-0891
Schiff, Manuel https://orcid.org/0000-0001-8272-232X
Funding for this research was provided by:
DIM Ile de France, DIM Thérapie génique
Article History
Received: 25 August 2020
Accepted: 17 May 2022
First Online: 7 June 2022
Competing interests
: F.M. and M.-G.B. are employees and equity holders of Spark Therapeutics, a Roche company. We declare that C.P., M.S.-S., P.d.L., C.O., M.C., and M.S. are designated as inventors of the European Patent Application EP20305079.4 filed on Jan. 29, 2020 in the names of Inserm, Fondation Imagine, Université de Paris, and APHP for “Gene therapy for MSUD.” The remaining authors declare no competing interests.