- A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation
[作者：Ramsey, BW; Davies, J; McElvaney, NG; Tullis, E; Bell, SC; Drevinek, P; Griese, M; McKone, EF; Wainwright, CE; Konstan, MW; Moss, R; Ratjen, F; Sermet-Gaudelus, I; Rowe, SM; Dong, QM; Rodriguez, S; Yen, K; Ordonez, C; Elborn, JS,期刊：New England Journal of Medicine, 页码：1663-1672 , 文章类型： Article，,卷期：2011年365-18]
- BackgroundIncreasing the activity of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein is a potential treatment for cystic fibrosis.MethodsWe conducted a randomized, double-blind, placebo-contr...
- Inducible Apoptosis as a Safety Switch for Adoptive Cell Therapy
[作者：Di Stasi, A; Tey, SK; Dotti, G; Fujita, Y; Kennedy-Nasser, A; Martinez, C; Straathof, K; Liu, E; Durett, AG; Grilley, B; Liu, H; Cruz, CR; Savoldo, B; Gee, AP; Schindler, J; Krance, RA; Heslop, HE; Spencer, DM; Rooney, CM; Brenner, MK,期刊：New England Journal of Medicine, 页码：1673-1683 , 文章类型： Article，,卷期：2011年365-18]
- BackgroundCellular therapies could play a role in cancer treatment and regenerative medicine if it were possible to quickly eliminate the infused cells in case of adverse events. We devised an inducible T-cell safety swi...
- Anti-Inhibitor Coagulant Complex Prophylaxis in Hemophilia with Inhibitors
[作者：Leissinger, C; Gringeri, A; Antmen, B; Berntorp, E; Biasoli, C; Carpenter, S; Cortesi, P; Jo, HJ; Kavakli, K; Lassila, R; Morfini, M; Negrier, C; Rocino, A; Schramm, W; Serban, M; Uscatescu, MV; Windyga, J; Zulfikar, B; Mantovani, L,期刊：New England Journal of Medicine, 页码：1684-1692 , 文章类型： Article，,卷期：2011年365-18]
- BackgroundPatients with severe hemophilia A and factor VIII inhibitors are at increased risk for serious bleeding complications and progression to end-stage joint disease. Effective strategies to prevent bleeding in such...
- Host and Pathogen Factors for Clostridium difficile Infection and Colonization
[作者：Loo, VG; Bourgault, AM; Poirier, L; Lamothe, F; Michaud, S; Turgeon, N; Toye, B; Beaudoin, A; Frost, EH; Gilca, R; Brassard, P; Dendukuri, N; Beliveau, C; Oughton, M; Brukner, I; Dascal, A,期刊：New England Journal of Medicine, 页码：1693-1703 , 文章类型： Article，,卷期：2011年365-18]
- BackgroundClostridium difficile infection is the leading cause of health care-associated diarrhea, and the bacterium can also be carried asymptomatically. The objective of this study was to identify host and bacterial fa...
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