Validation of a small molecule inhibitor of PDE6D-RAS interaction with favorable anti-leukemic effects von Canovas Nunes, Sara, De Vita, Serena, Anighoro, Andrew, Autelitano, François, Beaumont, Edward, Klingbeil, Pamela, McGuinness, Meaghan, Duvert, Beatrice, Harris, Chad, Yang, Lu, Pokharel, Sheela Pangeni, Chen, Chun-Wei, Ermann, Monika, Williams, David A., Xu, Haiming

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Stratifying Mortality Risk in Intensive Care: A Comprehensive Analysis Using Cluster Analysis and Classification and Regression Tree Algorithms von Romanelli, Antonio, Palmese, Salvatore, De Vita, Serena, Calicchio, Alessandro, Gammaldi, Renato

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Consensus-driven target product profiles for curative sickle cell disease gene therapies von Bukini, Daima, Makani, Julie, McCune, Joseph, Lee, Dennis, Bansbach, Cathy, De Vita, Serena, Kemps, Dominic, Amin, Elianna, Spector, Jonathan, Tisdale, John

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An NGF-responsive element targets myo-inositol monophosphatase-1 mRNA to sympathetic neuron axons von Andreassi, Catia, Zimmermann, Carola, Mitter, Richard, Fusco, Salvatore, De Vita, Serena, Saiardi, Adolfo, Riccio, Antonella

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Loss of function of TET2 cooperates with constitutively active KIT in murine and human models of mastocytosis von De Vita, Serena, Schneider, Rebekka K, Garcia, Michael, Wood, Jenna, Gavillet, Mathilde, Ebert, Benjamin L, Gerbaulet, Alexander, Roers, Axel, Levine, Ross L, Mullally, Ann, Williams, David A

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Loss‐of‐function JAK3 mutations in TMD and AMKL of Down syndrome von De Vita, Serena, Mulligan, Claire, McElwaine, Suzanne, Dagna‐Bricarelli, Franca, Spinelli, Monica, Basso, Giuseppe, Nizetic, Dean, Groet, Jürgen

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CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease von Sharma, Akshay, Boelens, Jaap-Jan, Cancio, Maria, Hankins, Jane S., Bhad, Prafulla, Azizy, Marjohn, Lewandowski, Andrew, Zhao, Xiaojun, Chitnis, Shripad, Peddinti, Radhika, Zheng, Yan, Kapoor, Neena, Ciceri, Fabio, Maclachlan, Timothy, Yang, Yi, Liu, Yi, Yuan, Jianping, Naumann, Ulrike, Yu, Vionnie W.C., Stevenson, Susan C., De Vita, Serena, LaBelle, James L.

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Secondary Ph+ acute lymphoblastic leukemia after temozolomide von De Vita, Serena, De Matteis, Silvia, Laurenti, Luca, Chiusolo, Patrizia, Reddiconto, Giovanni, Fiorini, Alessia, Leone, Giuseppe, Sica, Simona

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A Phase I Study of LSZ102, an Oral Selective Estrogen Receptor Degrader, with or without Ribociclib or Alpelisib, in Patients with Estrogen Receptor-Positive Breast Cancer von Jhaveri, Komal, Juric, Dejan, Yap, Yoon-Sim, Cresta, Sara, Layman, Rachel M, Duhoux, Francois P, Terret, Catherine, Takahashi, Shunji, Huober, Jens, Kundamal, Nicole, Sheng, Qing, Balbin, Alejandro, Ji, Yan, He, Wei, Crystal, Adam, De Vita, Serena, Curigliano, Giuseppe

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Brain exposure of the ATM inhibitor AZD1390 in humans-a positron emission tomography study von Jucaite, Aurelija, Stenkrona, Per, Cselényi, Zsolt, De Vita, Serena, Buil-Bruna, Nuria, Varnäs, Katarina, Savage, Alicia, Varrone, Andrea, Johnström, Peter, Schou, Magnus, Davison, Chris, Sykes, Andy, Pilla Reddy, Venkatesh, Hoch, Matthias, Vazquez-Romero, Ana, Moein, Mohammad Mahdi, Halldin, Christer, Merchant, Melinda S, Pass, Martin, Farde, Lars

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T-Charge™ Manufacturing of the Anti-BCMA CAR-T, Durcabtagene Autoleucel (PHE885), Promotes Expansion and Persistence of CAR-T Cells with High TCR Repertoire Diversity von Ikegawa, Shuntaro, Sperling, Adam S., Ansuinelli, Michela, Nikiforow, Sarah, Quinn, David, Bu, Dexiu, Mataraza, Jennifer, Pearson, David, Rispoli, Lawrence, Credi, Marc A, Orwitz, Nina, Arihara, Yohei, Prabhala, Rao, Potluri, Lakshmi Bhavani, Reynolds, Carol, Vita, Serena De, Munshi, Nikhil C, Ritz, Jerome

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Treatment of Individuals with Severe Sickle Cell Disease with OTQ923, an Autologous, Ex Vivo, CRISPR/Cas9-Edited, CD34+ Hematopoietic Stem and Progenitor Cell Product, Leads to Dur... von Sharma, Akshay, Boelens, Jaap Jan, Cancio, Maria I., Hankins, Jane, Bhad, Prafulla A, Lewandowski, Andrew, Zhao, Xiaojun, Chitnis, Shripad, Peddinti, Radhika, Zheng, Yan, Kapoor, Neena, Ciceri, Fabio, Yuan, Jianping, Yu, Vionnie W., Stevenson, Susan C., De Vita, Serena, LaBelle, James

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Small Molecule Inhibition of PDE6D-RAS Interaction Suppresses the Growth of Acute Lymphoblastic Leukemia Cell Lines von Canovas Nunes, Sara, Xu, Haiming, De Vita, Serena, Anighoro, Andrew, Autelitano, Francois, Beaumont, Edward, Klingbeil, Pamela, McGuinness, Meaghan, Duvert, Beatrice, Harris, Chad E., Yang, Lu, Pokharel, Sheela Pangeni, Chen, Chun-Wei David, Ermann, Monika, Williams, David A.

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Updated phase I study results of PHE885, a T-Charge manufactured BCMA-directed CAR-T cell therapy, for patients (pts) with r/r multiple myeloma (RRMM) von Sperling, Adam Samuel, Derman, Benjamin Avi, Nikiforow, Sarah, Im, Soo-Yeon, Ikegawa, Shuntaro, Prabhala, Rao H., Rodriguez, Diego Hernandez, Li, Yifang, Quinn, David S., Pearson, David, Bu, Dexiu, Mataraza, Jennifer, Liegel, Jessica, D'Souza, Anita, Rispoli, Lawrence, Credi, Marc, Ritz, Jerome, Jakubowiak, Andrzej J., De Vita, Serena, Munshi, Nikhil C.

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The gp130 cytokine IL-11 regulates engraftment of Vav1−/− hematopoietic stem and progenitor cells in lethally irradiated recipients von De Vita, Serena, Li, Yanhua, Harris, Chad E., McGuinness, Meaghan K., Ma, Clement, Williams, David A.

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Preclinical discovery and initial clinical data of WVT078, a BCMA × CD3 bispecific antibody von Raab, Marc S., Cohen, Yael C., Schjesvold, Fredrik, Aardalen, Kimberly, Oka, Adwait, Spencer, Andrew, Wermke, Martin, Souza, Anita D., Kaufman, Jonathan L., Cafro, Anna Maria, Ocio, Enrique M., Doki, Noriko, Henson, Kristin, Trabucco, Gina, Carrion, Ana, Bender, Florent C., Juif, Pierre-Eric, Fessehatsion, Adonai, Fan, Liqiong, Stonehouse, Jeffrey P., Blankenship, John W., Granda, Brian, De Vita, Serena, Lu, Haihui

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Acute promyelocytic leukemia in an HIV‐infected patient: A case report von Vita, Serena De, Matteis, Silvia De, Laurenti, Luca, Sorà, Federica, Tarnani, Michela, Cingolani, Antonella, Sica, Simona

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Abstract 4868: A preclinical PK/PD model based on a mouse glioblastoma survival model for AZD1390, a novel, brain-penetrant ATM kinase inhibitor, to predict the inhibition of DNA d... von Reddy, Venkatesh Pilla, Sykes, Andy, Colclough, Nicola, Durant, Stephen T., O'Connor, Lenka Oplustil, Hoch, Matthias, Bruna, Nuria Buil, Vita, Serena De, Merchant, Melinda, Pass, Martin

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Vav1, a Rho GTPase Guanine Exchange Factor (GEF) Is Required to Integrate gp130 Cytokine Signaling for Successful Engraftment of Murine Hematopoietic Stem Cells Following Irradiati... von De Vita, Serena, Li, Yanhua, Harris, Chad Everett, McGuinness, Meaghan, Williams, David A.

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Phase I Study of PHE885, a Fully Human BCMA-Directed CAR-T Cell Therapy for Relapsed/Refractory Multiple Myeloma Manufactured in <2 Days Using the T-Charge TM Platform von Sperling, Adam S., Nikiforow, Sarah, Nadeem, Omar, Mo, Clifton C, Laubach, Jacob P., Anderson, Kenneth C., Alonso, Alejandro, Ikegawa, Shuntaro, Prabhala, Rao, Hernandez Rodriguez, Diego, Daley, Heather, Shaw, Kit L., Arihara, Yohei, Ansari, Soudeh, Quinn, David S, Pearson, David, Hack, Anniesha, Treanor, Louise M, Bu, Dexiu, Mataraza, Jennifer, Rispoli, Lawrence, Credi, Marc, Ritz, Jerome, De Vita, Serena, Munshi, Nikhil C.

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