1. San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
2. Vita-Salute San Raffaele University, 20132 Milan, Italy.
3. IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.
4. Bioverativ, Waltham, MA 02451, USA.
5. GlaxoSmithKline R&D UK, Ware SG12 0DP, UK.
6. INSERM UMR1089, University of Nantes, CHU de Nantes, 44093 Nantes, France.