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

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 cytoreducti...

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Opis bibliograficzny
Główni autorzy: 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: Artigo
Język:Inglês
Wydane: American Society of Hematology 2012
Hasła przedmiotowe:
Plenary Paper
Dostęp online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3488882/
https://ncbi.nlm.nih.gov/pubmed/22968453
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2012-02-400937
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