Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans - Candotti, Fabio; Shaw, Kit L.; Muul, Linda; Carbonaro, Denise; Sokolic, Robert; Choi, Christopher; Schurman, Shepherd H.; Garabedian, Elizabeth; Kesserwan, Chimene; Jagadeesh, G. Jayashree; Fu, Pei-Yu; Gschweng, Eric; et al. (Blood, 2012)
Keywords: Adenosine Deaminase - deficiency; Adolescent; Agammaglobulinemia - therapy; Antigens, CD34 - metabolism; Biological and medical sciences; Bone Marrow Transplantation - methods; Child; Child, Preschool; Clinical Trials and Observations; Female; Gene Therapy; Genetic Therapy - methods; Genetic Vectors; Hematologic and hematopoietic diseases; Hematopoietic Stem Cell Transplantation - methods; Humans; Infant; Male; Medical sciences; Plenary Paper; Retroviridae - genetics; Severe Combined Immunodeficiency - therapy; Transduction, Genetic; Transplantation Conditioning; Young Adult; Virus; Immunopathology; Treatment; Hematology; Severe combined immunodeficiency; Enzyme; Retroviridae; Hydrolases; Adenosine deaminase; Gene therapy; Vector; Comparative study