Ontology highlight
ABSTRACT:
SUBMITTER: Candotti F
PROVIDER: S-EPMC3488882 | biostudies-literature | 2012 Nov
REPOSITORIES: biostudies-literature
Candotti Fabio F Shaw Kit L KL Muul Linda L Carbonaro Denise D Sokolic Robert R Choi Christopher C Schurman Shepherd H SH Garabedian Elizabeth E Kesserwan Chimene C Jagadeesh G Jayashree GJ Fu Pei-Yu PY Gschweng Eric E Cooper Aaron A Tisdale John F JF Weinberg Kenneth I KI Crooks Gay M GM Kapoor Neena N Shah Ami A Abdel-Azim Hisham H Yu Xiao-Jin XJ Smogorzewska Monika M Wayne Alan S AS Rosenblatt Howard M HM Davis Carla M CM Hanson Celine C Rishi Radha G RG Wang Xiaoyan X Gjertson David D Yang Otto O OO Balamurugan Arumugam A Bauer Gerhard G Ireland Joanna A JA Engel Barbara C BC Podsakoff Gregory M GM Hershfield Michael S MS Blaese R Michael RM Parkman Robertson R Kohn Donald B DB
Blood 20120911 18
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 and remained on ADA enzyme-replacement therapy (ERT) throughout the procedure. Only transient (months), low-level (< 0.01%) gene marking was observed in PBMCs of 2 older subjects (15 and 20 years of age ...[more]