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Journal Articles

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

Donald B. Kohn, MD, Kit L. Shaw, PhD, Elizabeth Garabedian, RN, MS, Denise Ann Carbonaro-Sarracino, PhD, Theodore B. Moore, MD, Satiro N. De Oliveira, MD, Gay M. Crooks, MD, John Tse, Sally Shupien, Dayna Terrazas, Alejandra Davila, Amalia Icreverzi, PhD, Allen Yu, Krista M. Chun, Christian E. Casas, Provaboti Barman, PhD, Maritess Coronel, Beatriz Campo Fernandez, PhD, Ruixue Zhang, Roger P. Hollis, PhD, Chilenwa Uzowuru, Hilory Ricketts, Jinhua Xu Bayford, Valentina Trevisan, Serena Arduini, PhD, Frances Lynn, Mahesh Kudari, MBBS, MA, Andrea Spezzi, MD MBBS, Lilith Reeves, MS, MT(ASCP), Kenneth Cornetta, Robert A. Sokolic, MD, Roberta Parrott, BS, Rebecca Buckley, MD, Claire Booth, Fabio Candotti, MD, Harry L. Malech, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, H. Bobby Gaspar, MBBS, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3345.
https://doi.org/10.1182/blood-2019-123432
Published: 2019
Journal Articles

Phase I Study of the PTEFb Inhibitor BAY 1251152 in Patients with Acute Myelogenous Leukemia

Michael Byrne, MD, Mark G. Frattini, MD PhD, Oliver G. Ottmann, MD, Ioannis Mantzaris, MD MS, Martin Wermke, MD, Daniel J. Lee, MD, Daniel Morillo, MD, Arne Scholz, PhD, Stuart Ince, PhD, Ray Valencia, MD, Fabricio Souza, MD, Raul Cordoba, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 4055.
https://doi.org/10.1182/blood-2018-99-117257
Published: 2018
Journal Articles

CDK9 Inhibition with LDC526 Is an Effective Treatment for T-Cell Prolymphocytic Leukemia (T-PLL) in Vitro

Patricia Johansson, MD, Peter Habenberger, PhD, Ludger Klein-Hitpass, PhD, Ulrich Duehrsen, MD, Ralf Küppers, PhD, Carsten Schultz-Fademrecht, PhD, Axel Choidas, PhD, Jan Dürig, MD
Journal: Blood
Blood (2017) 130 (Supplement 1): 1262.
https://doi.org/10.1182/blood.V130.Suppl_1.1262.1262
Published: 2017
Journal Articles

The CDK7 Inhibitor THZ1 Alters RNA Polymerase Dynamics at the 5' and 3' Ends of Genes

Shilpa Sampathi, PhD, Pankaj Acharya, PhD, Yue Zhao, PhD, Jing Wang, PhD, Kristy Stengel, PhD, Qi Liu, PhD, Michael R. Savona, MD, Scott Hiebert, PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 1195.
https://doi.org/10.1182/blood.V130.Suppl_1.1195.1195
Published: 2017
Journal Articles

Randomized Phase III Study (ADMYRE) of Plitidepsin in Combination with Dexamethasone Vs. Dexamethasone Alone in Patients with Relapsed/Refractory Multiple Myeloma

Ivan Spicka, MDPhD, Enrique M. Ocio, MDPhD, Heather E. Oakervee, MBBS MD, Richard Greil, MD, Raymond H. Banh, MD, Laurence Catley, MD, Shang-Yi Huang, MD, PhD, James M. D'Rozario, MBBS FRACP FRCPA, Meletios A. Dimopoulos, José Rodríguez, MDPhD, Sara Martinez, MD, Sonia Extremera, BsC, Vicente Alfaro, MsC PhD, Angelo Michele Carella, MD, Nathalie Meuleman, MDPhD, Roman Hájek, MD, Argiris Symeonidis, MD PhD, Chang-Ki Min, MD, Paul Cannell, MD, Heinz Ludwig, MD, Pieter Sonneveld, MDPhD, Maria V. Mateos, MDPhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 1886.
https://doi.org/10.1182/blood.V130.Suppl_1.1886.1886
Published: 2017
Journal Articles

Epigenetic and Transcriptomic Profiling of MLL Rearranged Infant Acute Lymphoblastic Leukemia

Tomoya Isobe, Aiko Sato, Kenichi Yoshida, MD PhD, Chika Kobayashi, Yuichi Shiraishi, PhD, Kenichi Chiba, B.A., Hiroko Tanaka, B.A., Shiro Fukuda, Shogo Yamamoto, Kenji Tatsuno, Shuichi Tsutsumi, Nobutaka Kiyokawa, MD PhD, Yuki Aoki, Takashi Ishihara, Daisuke Tomizawa, MD PhD, Takako Miyamura, MD PhD, Eiichi Ishii, MD PhD, Satoru Miyano, PhD, Seishi Ogawa, MD PhD, Hiroyuki Aburatani, Shuki Mizutani, Junko Takita, MD PhD, Masatoshi Takagi, MD PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 3809.
https://doi.org/10.1182/blood.V130.Suppl_1.3809.3809
Published: 2017
Journal Articles

Targeting the Transcriptional Addiction of Leukemia Stem Cells By a New Class of Protein Kinase Inhibitors

Yinon Ben-Neriah, MD PhD, Avanthika Venkatachalam, MSc, Avner Fink, PhD, Eric Hung, MSc, Joseph Vacca, PhD, Frank Mercurio, PhD, Moshe Oren, PhD, Eli Pikarsky, MDPhD, Irit Snir-Alkalay, PhD, Waleed Minzel, MSc
Journal: Blood
Blood (2017) 130 (Supplement 1): 812.
https://doi.org/10.1182/blood.V130.Suppl_1.812.812
Published: 2017
Journal Articles

Selective Targeting Cyclin-Dependent Kinase-9 (CDK9) Downmodulates c-MYC and Induces Apoptosis in Diffuse Large B-Cell Lymphoma (DLBCL) Cells

Taylor Rowland, Cody Paiva, BS, Joelle Rowley, Andy Chen, MD PhD, Lisa Drew, PhD, Peter Hurlin, Bill H. Chang, MD PhD, Alexey V Danilov, MD PhD
Journal: Blood
Blood (2016) 128 (22): 289.
https://doi.org/10.1182/blood.V128.22.289.289
Published: 2016
Journal Articles

Inhibition of Cyclin Dependent Kinase 9 Synergistically Enhanced Venetoclax Activity in Mantle Cell Lymphoma Cells

Xiaoxian Zhao, PhD, Juraj Bodo, PhD, Ruoying Chen, PhD, Lisa Durkin, Andrew J. Souers, PhD, Darren C. Phillips, PhD, Eric D. Hsi, MD
Journal: Blood
Blood (2016) 128 (22): 1593.
https://doi.org/10.1182/blood.V128.22.1593.1593
Published: 2016
Journal Articles

Lentiviral Vectors Incorporating Ubiquitous Chromatin Opening Element Driving Canine CD18 Expression

Everette J. R. Nelson, PhD, Laura M. Tuschong, B.S., Dennis D. Hickstein, MD
Journal: Blood
Blood (2016) 128 (22): 5890.
https://doi.org/10.1182/blood.V128.22.5890.5890
Published: 2016
Journal Articles

An IGF2BP3-Cdk9 Pathway Governs the Human Fetal-Adult Megakaryocyte Transition

Kamaleldin E Elagib, MBBS, PhD, Chih-Huan Lu, BS, Goar Mosoyan, Ewelina Zasadzińska, MS, Daniel R Foltz, PhD, Peter Balogh, Els Verhoeyen, Miriam Sansó, PhD, Robert P Fisher, MD PhD, Camelia Iancu-Rubin, PhD, Adam N Goldfarb, MD
Journal: Blood
Blood (2016) 128 (22): 886.
https://doi.org/10.1182/blood.V128.22.886.886
Published: 2016
Journal Articles

Bayesian Network Models of Multiple Myeloma: Drivers of High Risk and Durable Response

Fred Gruber, PhD, Jonathan J Keats, PhD, Kyle McBride, MS, Karl Runge, PhD, Diane Wuest, Tiffany Hadzi, Mary Derome, MS, Sagar Lonial, MD FACP, Iya Khalil, PhD, Boris Hayete, Daniel Auclair, PhD
Journal: Blood
Blood (2016) 128 (22): 4406.
https://doi.org/10.1182/blood.V128.22.4406.4406
Published: 2016
Journal Articles

Transcription Pausing Regulates Hematopoietic Stem Cell Emergence through Fine-Tuning of Signaling Pathways

Qiwen Yang, PhD, Xiuli Liu, PhD, Ting Zhou, PhD, Jennifer Cook, Kim Nguyen, Xiaoying Bai, PhD
Journal: Blood
Blood (2015) 126 (23): 2379.
https://doi.org/10.1182/blood.V126.23.2379.2379
Published: 2015
Journal Articles

The Molecular Mechanism of Transcriptional Activation By MLL-AEP Fusion Proteins

Akihiko Yokoyama, PhD, Hiroshi Okuda, PhD
Journal: Blood
Blood (2015) 126 (23): 2435.
https://doi.org/10.1182/blood.V126.23.2435.2435
Published: 2015
Journal Articles

Lentiviral Hematopoietic Stem Cell Gene Therapy for Older Patients with X-Linked Severe Combined Immunodeficiency

Suk See De Ravin, MDPhD, Xiaolin Wu, Narda Theobald, Janet S Lee, John Gray, PhD, Imelda Celine Hanson, MD, Luigi Notarangelo, MDPhD, Brian P. Sorrentino, MD, Harry L. Malech, MD
Journal: Blood
Blood (2015) 126 (23): 261.
https://doi.org/10.1182/blood.V126.23.261.261
Published: 2015
Journal Articles

Human Induced Pluripotent Stem Cells Incorporating Safe Harbor Loci Integrated Inducible Suicide Systems for Use in the Application of Cellular Therapeutics

Xiaosong Huang, PhD, Weijie Lan, PhD, Philippe Parone, PhD, Raedun Clarke, PhD, Ramzey Abujarour, PhD, Megan Robinson, BS, Miguel Meza, MS, Sarah Raynel, MS, Betsy Rezner, BS, David Robbins, BS, Tom Tong Lee, PhD, Daniel Shoemaker, PhD, Bahram Valamehr, PhD MBA
Journal: Blood
Blood (2015) 126 (23): 3237.
https://doi.org/10.1182/blood.V126.23.3237.3237
Published: 2015
Journal Articles

Pgk-Mediated Expression of Common Gamma Chain Is More Effective Than EF1a for Therapeutic Immune Reconstitution of X-SCID Dogs after In Vivo Gene Therapy with Foamy Virus Vector

Olivier Humbert, PhD, Christopher R Burtner, PhD, Patricia O'Donnell, Daniel R Humphrys, B.S., Nicholas Hubbard, Frieda Chan, PhD, Jennifer E Adair, PhD, Grant D. Trobridge, PhD, Troy Torgerson, MD PhD, Andrew M. Scharenberg, MD, David J. Rawlings, MD, Peter Felsburg, Hans-Peter Kiem, MD
Journal: Blood
Blood (2015) 126 (23): 262.
https://doi.org/10.1182/blood.V126.23.262.262
Published: 2015
Journal Articles

Single-Cell-State Culture of Human Pluripotent Stem Cells Increases Transfection Efficiency

Takenobu Nii, PhD, Hiroshi Kohara, PhD, Tomotoshi Marumoto, MD PhD, Tetsushi Sakuma, PhD, Takashi Yamamoto, PhD, Kenzaburo Tani, MD PhD
Journal: Blood
Blood (2015) 126 (23): 2037.
https://doi.org/10.1182/blood.V126.23.2037.2037
Published: 2015
Journal Articles

Robust Therapeutic Expression of the Common Gamma Chain with the Human Pgk Promoter Using Foamy Virus in Vivo Gene Therapy in a Canine Model of Severe Combined Immunodeficiency

Christopher R Burtner, PhD, Olivier Humbert, PhD, Patricia O'Donnell, Nicholas Hubbard, Daniel Humphrys, Jennifer E Adair, PhD, Grant D Trobridge, PhD, Troy R Torgerson, MD PhD, Andrew M Scharenberg, MD, David Rawlings, MD, Peter J Felsburg, VMD, PhD, Hans-Peter Kiem, MD
Journal: Blood
Blood (2014) 124 (21): 4794.
https://doi.org/10.1182/blood.V124.21.4794.4794
Published: 2014
Journal Articles

Mechanistic Role of HEXIM1 Induction in BRD4-Antagonist Mediated Growth Inhibition, Differentiation and in Vivo Lethal Activity Against Human AML Blast Progenitor Cells

Santhana G. T. Devaraj, PhD, Warren Fiskus, PhD, Bhavin Shah, Sunil Sharma, Jun Qi, PhD, Baohua Sun, MD PhD, Saikat Saha, PhD, Swaminathan Padmanabhan Iyer, MD, Youli Zu, MD PhD, James Bradner, MD, Kapil N. Bhalla, MD
Journal: Blood
Blood (2014) 124 (21): 3534.
https://doi.org/10.1182/blood.V124.21.3534.3534
Published: 2014