Long-term safety and correction of immune and metabolic defects in ADA-SCID children treated with gene therapy

Aiuti, A; Cassani, B; Benninghoff, U; Cattaneo, F; Callegaro, L; Scaramuzza, S; Andolfi, G; Brigida, I; Mirolo, M; Tabucchi, A; Carlucci, F; Eibl, M; Aker, M; Slavin, S; Galimberti, S; Valsecchi, Mg; Chiesa, R; Marktel, S; Ciceri, Fabio; Miniero, R; Bordignon, Claudio; Roncarolo, Mg