Gene Therapy for “Bubble Boy” Disease

Cell

Volumn 166, Issue 2, 2016, Pages 263-

  Hoggatt, Jonathan ^a  

^a MASSACHUSETTS GENERAL HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOSINE DEAMINASE; CD34 ANTIGEN; GENE THERAPY AGENT; STRIMVELIS; TOXIN; UNCLASSIFIED DRUG;

ADENOSINE DEAMINASE DEFICIENCY; BUBBLE BOY DISEASE; GENE THERAPY; HLA MATCHING; HUMAN; IMMUNOCOMPETENT CELL; MORBIDITY; PHARMACEUTICAL CARE; PRIORITY JOURNAL; SEVERE COMBINED IMMUNODEFICIENCY; SHORT SURVEY;

EID: 84982150687     PISSN: 00928674     EISSN: 10974172     Source Type: Journal    
DOI: 10.1016/j.cell.2016.06.049     Document Type: Short Survey

References (3)

1. Aiuti, A., Slavin, S., Aker, M., Ficara, F., Deola, S., Mortellaro, A., Morecki, S., Andolfi, G., Tabucchi, A., Carlucci, F., et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 296 (2002), 2410–2413.
2. Aiuti, A., Cattaneo, F., Galimberti, S., Benninghoff, U., Cassani, B., Callegaro, L., Scaramuzza, S., Andolfi, G., Mirolo, M., Brigida, I., et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N. Engl. J. Med. 360 (2009), 447–458.
3. Cicalese, M.P., Ferrua, F., Castagnaro, L., Pajno, R., Barzaghi, F., Giannelli, S., Dionisio, F., Bonopane, M., Casiraghi, M., Tabucchi, A., et al. Blood, 2016, 10.1182/blood-2016-01-688226 Published online April 29, 2016.