Experimental Gene Therapy Frees ‘Bubble-boy’ Babies From a Life of Isolation
22 Apr, 2019 | 01:48h | UTCExperimental gene therapy frees ‘bubble-boy’ babies from a life of isolation – Nature (free)
Original Article: Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1 – New England Journal of Medicine (link to abstract – $ for full-text)