title: Gene therapy for ADA-SCID, the first marketing approval of anex vivogene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products.
authors.affiliation: San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.|Vita Salute San Raffaele University, Milan, Italy.
authors.forename: Alessandro
authors.identifier: 0000-0002-5398-1717
authors.initials: A
authors.lastname: Aiuti
authors.affiliation: San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.|Vita Salute San Raffaele University, Milan, Italy.
authors.forename: Maria Grazia
authors.initials: MG
authors.lastname: Roncarolo
authors.affiliation: San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, Italy.|Vita Salute San Raffaele University, Milan, Italy.
authors.forename: Luigi
authors.initials: L
authors.lastname: Naldini
mesh_terms: D000243:Adenosine Deaminase / Q000172:deficiency* / Q000235:genetics; D000361:Agammaglobulinemia / Q000235:genetics / Q000628:therapy*; D002648:Child; D005060:Europe; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000627:therapeutic use; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D016511:Severe Combined Immunodeficiency / Q000235:genetics / Q000628:therapy*; D014182:Transplantation, Autologous
chemical_list: C548482:ADA protein, human; D000243:Adenosine Deaminase