1. San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), IRCSS Ospedale San Raffaele, Milan, Italy
2. DNA and RNA Medicine Division, CIMA, Universidad de Navarra, IdisNA, Pamplona, Spain
3. University of Rouen, INSERM, U1234, Pathophysiology Autoimmunity and Immunotherapy (PANTHER), Normandie University, Rouen, France
4. Vivet Therapeutics S.L., Pamplona, Spain; and
5. Spark Therapeutics, Inc., Philadelphia, Pennsylvania, USA.