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Pulmonary Cell NewsScientists treated 50 patients with Severe combined immunodeficiency due to adenosine deaminase (ADA) with an investigational gene therapy composed of autologous CD34+ hematopoietic stem and progenitor cells transduced ex vivo with a self-inactivating lentiviral vector encoding human ADA.
[New England Journal of Medicine]
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Kohn, D. B., Booth, C., Shaw, K. L., Xu-Bayford, J., Garabedian, E., Trevisan, V., Carbonaro-Sarracino, D. A., Soni, K., Terrazas, D., Snell, K., Ikeda, A., Leon-Rico, D., Moore, T. B., Buckland, K. F., Shah, A. J., Gilmour, K. C., De Oliveira, S., Rivat, C., Crooks, G. M., … Gaspar, H. B. (2021). Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency. New England Journal of Medicine, 0(0), null. https://doi.org/10.1056/NEJMoa2027675 Cite
