Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans

• Published in: Blood Vol. 120; no. 18; pp. 3635 - 3646
• Main Authors: 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, Cooper, Aaron, Tisdale, John F., Weinberg, Kenneth I., Crooks, Gay M., Kapoor, Neena, Shah, Ami, Abdel-Azim, Hisham, Yu, Xiao-Jin, Smogorzewska, Monika, Wayne, Alan S., Rosenblatt, Howard M., Davis, Carla M., Hanson, Celine, Rishi, Radha G., Wang, Xiaoyan, Gjertson, David, Yang, Otto O., Balamurugan, Arumugam, Bauer, Gerhard, Ireland, Joanna A., Engel, Barbara C., Podsakoff, Gregory M., Hershfield, Michael S., Blaese, R. Michael, Parkman, Robertson, Kohn, Donald B.
• Format: Journal Article
• Language: English
• Published: Washington, DC Elsevier Inc 01.11.2012; Americain Society of Hematology; American Society of Hematology
• Subjects: 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

We conducted a gene therapy trial in 10 patients with adenosine deaminase (ADA)–deficient severe combined immunodeficiency using 2 slightly different retroviral vectors for the transduction of patients' bone marrow CD34+ cells. Four subjects were treated without pretransplantation cytoreduction...