Blood

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency.

Blood

Cicalese MP, Ferrua F, Castagnaro L, Pajno R, Barzaghi F, Giannelli S, Dionisio F, Brigida I, Bonopane M, Casiraghi M, Tabucchi A, Carlucci F, Grunebaum E, Adeli M, Bredius RG, Puck JM, Stepensky P, Tezcan I, Rolfe K, De Boever E, Reinhardt RR, Appleby J, Ciceri F, Roncarolo MG, Aiuti A

Normal ABL1 is a tumor suppressor and therapeutic target in human and mouse leukemias expressing oncogenic ABL1 kinases.

Blood

Dasgupta Y, Koptyra M, Hoser G, Kantekure K, Roy D, Gornicka B, Nieborowska-Skorska M, Bolton-Gillespie E, Cerny-Reiterer S, Müschen M, Valent P, Wasik MA, Richardson C, Hantschel O, van der Kuip H, Stoklosa T, Skorski T

Normal ABL1 is a tumor suppressor and therapeutic target in human and mouse leukemias expressing oncogenic ABL1 kinases.

Blood

Dasgupta Y, Koptyra M, Hoser G, Kantekure K, Roy D, Gornicka B, Nieborowska-Skorska M, Bolton-Gillespie E, Cerny-Reiterer S, Müschen M, Valent P, Wasik MA, Richardson C, Hantschel O, van der Kuip H, Stoklosa T, Skorski T

Identifying Drug-Resistant Mutations in Ebf1-Pdgfrb Ph-like Acute Lymphoblastic Leukemia.

Blood

Thai Hoa Tran, Jonathan Van Nguyen, Catherine C. Smith, Kathryn G. Roberts, Charles G. Mullighan, Neil P. Shah, Mignon L. Loh

Recurrent Mutations in CCND3 Confer Clinical Resistance to FLT3 Inhibitors.

Blood

Catherine C. Smith, Aaron D. Viny, Evan S. Massi, Cyriac Kandoth, Nicholas D. Socci, Henry Hsu, Brian West, Gideon Bollag, Barry S. Taylor, Ross L. Levine, Neil P. Shah

Pharmacokinetic Profile and Pharmacodynamic Effects of ASP2215, a Selective, Potent Inhibitor of FLT3/AXL, in Patients with Relapsed or Refractory Acute Myeloid Leukemia: Results from a First-in-Human Phase 1/2 Study.

Blood

Catherine C. Smith, Mark J. Levis, Mark R Litzow, Alexander E. Perl, Jessica K. Altman, Stanley C. Gill, Takeshi Kadokura, Geoffrey J. Yuen, Ogert Fisniku, Charles Liu, Itsuro Nagase, Briana Sargent, Noreen Welter, Erkut Bahceci

Expression of B and T Lymphocyte Attenuator (BTLA) Correlates with CNS Metastasis and Adverse Prognosis in Activated B-Cell Lymphoma and Acute Lymphoblastic Leukemia.

Blood

Huimin Geng, Zhengshan Chen, Shawn Anderson, Eleanor Fraser, Ming Lu, Chen Lingjing, Colin Collins, Muschen Markus, James L. Rubenstein

Targeted Activation of B Cell Autoimmunity Checkpoints in Acute Lymphoblastic Leukemia.

Blood

Zhengshan Chen, Huimin Geng, Clifford A. Lowell, Arthur Weiss, Stephen P. Hunger, Ari Melnick, Markus Muschen

Ibrutinib Significantly Prolonged Survival in a Human Burkitt Lymphoma (BL) Xenograft NSG Mouse Model: Ibrutinib May be a Potential Adjuvant Agent in the Treatment of BL.

Blood

Sanghoon Lee, Changhong Yin, Janet Ayello, Erin Morris, Timmy O'Connell, Lauren Harrison, Carmella van de Ven, Matthew J. Barth, Rodney R. Miles, Paul J. Galardy, Stanton Goldman, Megan S Lim, Michelle L. Hermiston, Linda McAllister-Lucas, Lisa Giulino-Roth, Sherrie L. Perkins, Mitchell S. Cairo

Ibrutinib Alone and in Combination with Dexamethasone and Carfilzomib Significantly Inhibits Cell Proliferation in Primary Mediastinal B-Cell Lymphoma (PMBL): Ibrutinib May be a Future Targeted Agent in Combination Therapy in Patients with PMBL.

Blood

Sanghoon Lee, Changhong Yin, Janet Ayello, Erin Morris, Lauren Harrison, Carmella van de Ven, Matthew J. Barth, Rodney R. Miles, Paul J. Galardy, Stanton Goldman, Megan S Lim, Michelle L. Hermiston, Linda McAllister-Lucas, Lisa Giulino-Roth, Sherrie L. Perkins, Mitchell S. Cairo

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