Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

BACKGROUND: Severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a rare and life-threatening primary immunodeficiency. METHODS: We treated 50 patients with ADA-SCID (30 in the United States and 20 in the United Kingdom) with an investigational gene therapy compo...

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Bibliografske podrobnosti
izdano v:N Engl J Med
Main Authors: 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., Izotova, N., Tse, J., Adams, S., Shupien, S., Ricketts, H., Davila, A., Uzowuru, C., Icreverzi, A., Barman, P., Campo Fernandez, B., Hollis, R.P., Coronel, M., Yu, A., Chun, K.M., Casas, C.E., Zhang, R., Arduini, S., Lynn, F., Kudari, M., Spezzi, A., Zahn, M., Heimke, R., Labik, I., Parrott, R., Buckley, R.H., Reeves, L., Cornetta, K., Sokolic, R., Hershfield, M., Schmidt, M., Candotti, F., Malech, H.L., Thrasher, A.J., Gaspar, H.B.
Format: Artigo
Jezik:Inglês
Izdano: 2021
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Article
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC8240285/
https://ncbi.nlm.nih.gov/pubmed/33974366
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa2027675
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