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

@article{Candotti2012GeneTF,
  title={Gene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.},
  author={Fabio Candotti and Kit L. Shaw and Linda Mesler Muul and Denise A. Carbonaro and Robert A Sokolic and Christopher Choi and Shepherd H. Schurman and Elizabeth K Garabedian and Chimene A Kesserwan and G Jayashree Jagadeesh and Pei-Yu Fu and Eric H. Gschweng and Aaron Cooper and John F. Tisdale and Kenneth I. Weinberg and Gay M. Crooks and Neena Kapoor and Ami Shah and Hisham Abdel-Azim and Xiao-jin Yu and Monika Smogorzewska and Alan S. Wayne and Howard M. Rosenblatt and Carla M Davis and Celine I. Hanson and Radha G Rishi and Xiaoyan Wang and David Gjertson and Otto O Yang and Arumugam Balamurugan and Gerhard Bauer and Joanna A Ireland and Barbara C Engel and Gregory M. Podsakoff and Michael S. Hershfield and R. Michael Blaese and Robertson Parkman and Donald B Kohn},
  journal={Blood},
  year={2012},
  volume={120 18},
  pages={3635-46}
}
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… CONTINUE READING
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Molecular follow-up of the German WAS Clinical Gene Therapy Trial

  • A Paruzynski, K Boztug, C Ball
  • Paper presented at: 14th Annual Meeting of the…
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