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Journal Articles

Introduction to a review series on gene therapy and gene editing for sickle cell disease and hemophilia

Free
Catherine M. Bollard
Journal: Blood
Blood (2021) 138 (11): 913.
https://doi.org/10.1182/blood.2021012891
Published: 2021
Journal Articles

Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells

Free
Suk See De Ravin, Julie Brault, Ronald J. Meis, Siyuan Liu, Linhong Li, Mara Pavel-Dinu, Cicera R. Lazzarotto, Taylor Liu, Sherry M. Koontz, Uimook Choi, Colin L. Sweeney, Narda Theobald, GaHyun Lee, Aaron B. Clark, Sandra S. Burkett, Benjamin P. Kleinstiver, Matthew H. Porteus, Shengdar Tsai, Douglas B. Kuhns, Gary A. Dahl, Stephen Headey, Xiaolin Wu, Harry L. Malech
Journal: Blood
Blood (2021) 137 (19): 2598–2608.
https://doi.org/10.1182/blood.2020008503
Published: 2021
Includes: Supplemental data
Journal Articles

Identification of Novel Variants Associated with Fetal Hemoglobin Levels in Healthy Donors (the INTERVAL study)

Free
Emily Kawabata, Samuel Lessard, PhD, Dirk Paul, Paola G Bronson, Robert Peters, Sriram Krishnamoorthy, PhD, William Astle, Adam Butterworth
Journal: Blood
Blood (2019) 134 (Supplement_1): 2243.
https://doi.org/10.1182/blood-2019-123977
Published: 2019
Journal Articles

CRISPR/dCas9 (D10A) -Mediated Hb CS Gene Editing and Identification of Genetically Modified Fibroblasts

Free
Lian Yu, Wei Hao Wu, WANG Shunqing, Ken H. Young, Funeng Jiang, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 5754.
https://doi.org/10.1182/blood-2019-129931
Published: 2019
Journal Articles

Fanconi Anemia: A Paradigm for Understanding DNA Repair During Replication.

Free
Agata Smogorzewska, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): SCI-32.
https://doi.org/10.1182/blood-2019-121229
Published: 2019
Journal Articles

CC-90009, a Novel Cereblon E3 Ligase Modulator, Targets GSPT1 for Degradation to Induce Potent Tumoricidal Activity Against Acute Myeloid Leukemia (AML)

Free
Antonia Lopez-Girona, Gang Lu, Emily Rychak, Derek Mendy, Chin-Chun Lu, Irit Rappley, Celia Fontanillo, Brian E. Cathers, Thomas O. Daniel, Joshua Hansen
Journal: Blood
Blood (2019) 134 (Supplement_1): 2703.
https://doi.org/10.1182/blood-2019-127892
Published: 2019
Journal Articles

Efficacy Proof of Concept for Allogeneic CD123 Targeting CAR T-Cells Against Primary Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN): Efficient Control of Tumor Progression in PDX Model and Potential Loss of CD123 Expression in Relapsed Disease

Free
Tianyu Cai, PhD, Kathryn L Black, PhD, Ammar S. Naqvi, PhD, Deanne Taylor, PhD, Ming Zhao, MD, Qi Yuan, Mayumi Sugita, MD, Roman Galetto, PhD, Agnès Gouble, PhD, Julianne N. P. Smith, PhD, Antonio Cavazos, Lina Han, PhD, Qi Zhang, PhD, Vinitha Mary Kuruvilla, MSc, Helen Ma, MS, Sergej Konoplev, Guilin Tang, MD, Jun Gu, MD PhD, Xiaoping Su, PhD, Sattva S Neelapu, MD, Andrew A. Lane, MD PhD, Hagop M. Kantarjian, MD, Monica L. Guzman, PhD, Naveen Pemmaraju, MD, Andrei Thomas-Tikhonenko, PhD, Marina Y Konopleva, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2659.
https://doi.org/10.1182/blood-2019-126869
Published: 2019
Journal Articles

ASXL1 Mutations Are Associated with Widespread and Distinct DNA Methylation Alterations

Free
Nora E Rahmani, MD, Nandini Ramachandra, Tushar D Bhagat, PhD, Shanisha Gordon, Kith Pradhan, PhD, Bianca Rivera Pena, Mohammad Kazemi, Orsi Giricz, PhD, Shahina Maqbool, Raul Olea, Hamid Dolatshad, Vickram Tittrea, Dharamveer Tatwavedi, Shalini Singh, Andrea Pellagatti, Jacqueline Boultwood, Amit Verma, MBBS
Journal: Blood
Blood (2019) 134 (Supplement_1): 2989.
https://doi.org/10.1182/blood-2019-126850
Published: 2019
Journal Articles

Identification of Genetic Vulnerabilities and Synthetic-Lethal Targets in NSD2-High Multiple Myeloma

Free
Amin Sobh, PhD, Charlotte Kaestner, Alberto Riva, PhD, Jonathan D. Licht, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3757.
https://doi.org/10.1182/blood-2019-125951
Published: 2019
Journal Articles

A Gain of Function Mutation in the NSD2 Histone Methyltransferase Drives Glucocorticoid Resistance Via Blocking Receptor Auto-Induction and BIM/Bmf Expression in ALL

Free
Jianping Li, MD, Catalina Troche, PhD, Julia Hlavka Zhang, BS, Jonathan Shrimp, PhD, Jacob S. Roth, PhD, Duohui Jing, PhD, Alok Swaroop, PhD, Marta Kulis, Jon Oyer, PhD, Christine Will, MS, Daphne Dupere-Richer, PhD, Alberto Riva, PhD, Crissandra Pipe, LATG, Amin Sobn, PhD, Sharon Norton, MS, Richard Lynn Bennett, PhD, Min Shen, PhD, Matthew D. Hall, PhD, Richard B. Lock, PhD, Jonathan D. Licht, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3758.
https://doi.org/10.1182/blood-2019-126910
Published: 2019
Journal Articles

An Authentic Murine Model of Fetal/Neonatal Alloimmune Thrombocytopenia

Free
Huiying Zhi, PhD, Maria Therese Ahlen, PhD, Trude Rasmussen, Bjørn Skogen, MD, Peter J. Newman, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 97.
https://doi.org/10.1182/blood-2019-130573
Published: 2019
Journal Articles

Germline Mutations Predispose to Familial Myeloproliferative Neoplasms

Free
Stephanie Berg, DO, Manuel O. Diaz, MD, Sucha Nand, MD, Jiwang Zhang, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2969.
https://doi.org/10.1182/blood-2019-124805
Published: 2019
Journal Articles

Interrogating the Role of ASXL1 and JAK2 mutations in Myeloproliferative Neoplasms Utilizing Human Pluripotent Stem Cells

Free
Taylor B. Collins, Stephanie A. Luff, PhD, Luis F.Z. Batista, PhD, Christopher M. Sturgeon, PhD, Stephen T. Oh, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2971.
https://doi.org/10.1182/blood-2019-130541
Published: 2019
Journal Articles

Allogeneic Anti-CD19 CAR T Cells Manufactured from Healthy Donors Provide a Unique Cellular Product with Distinct Phenotypic Characteristics Compared to CAR T Cells Generated from Patients with Mature B Cell Malignancies

Free
Charlotte Graham, MRCP, BSc, MBBS, Agnieszka Jozwik, PhD, Ruby Quartey-Papafio, Nikolaos Ioannou, PhD MSc, BSc, Ana M Metelo, PhD, Carlo Scala, PhD, Glenda Dickson, PhD, Orla Stewart, Maria Almena-Carrasco, Elisa Peranzoni, Alan G. Ramsay, BSc,MSc,PhD, Sandra Dupouy, Pharm.D, PhD, Farzin Farzaneh, PhD, Piers E.M. Patten, BSc, MRCP, MRCPath, PhD, Andrea Pepper, PhD, Reuben Benjamin, FRCPath, MRCP, MBBS, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3228.
https://doi.org/10.1182/blood-2019-123018
Published: 2019
Journal Articles

Manufacture and In Vivo Support of Gene-Edited, Regulatory-like, T-Cells (edTreg) Using a Synthetic IL2 Receptor

Free
Gene I. Uenishi, PhD, Cornell Mallari, Luis Gamboa, Samuel West, Chester Jacobs, Noelle Dahl, Greg Boucher, Samantha Chin, Rangoli Aeran, Dariusz Wodziak, Simon Gebremeskel, Karen Vo, PhD, Cade E. Ito, Laura Smith, Chandra Patel, PhD, Keith Abe, PhD, Basha Stankovich, PhD, Karen Sommer, PhD, Troy R. Torgerson, MD PhD, David J. Rawlings, MD, Andrew M. Scharenberg, MD, Greg J. Cost, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3355.
https://doi.org/10.1182/blood-2019-130945
Published: 2019
Journal Articles

Primary Human NK Cell Gene-Editing Reveals a Critical Role for NKG2A in Cytokine-Induced Memory-like NK Cell Responses

Free
Melissa M Berrien-Elliott, PhD, Wong Pamela, B.S., Carly Neal, B.S., Julia A Wagner, PhD, Michelle Becker-Hapak, BS, Tim Schappe, BS, Matthew L. Cooper, PhD, Emily M Mace, PhD, Todd A Fehniger, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3237.
https://doi.org/10.1182/blood-2019-129162
Published: 2019
Journal Articles

Fully Closed, Large-Scale, and Clinical Grade Cell Sorting of Hematopoietic Stem Cell (HSC)-Enriched CD90 + Cells for Transplantation and Gene Therapy

Free
Stefan Radtke, PhD, Margaret Cui, Anai M Perez, Yan-Yi Chan, Stefanie Schmuck, BS, Andrew J Berger, PhD, Thomas Eunson, BSc, Hans-Peter Kiem, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3246.
https://doi.org/10.1182/blood-2019-125969
Published: 2019
Journal Articles

NK Cells Lacking CD38 Are Resistant to Oxidative Stress-Induced Death

Free
Frank Cichocki, PhD, Karrune Woan, MD PhD, Cheng-Ying Wu, Bruce R. Blazar, MD, Ryan Bjordahl, PhD, Bahram Valamehr, PhD, Jeffrey S. Miller, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3215.
https://doi.org/10.1182/blood-2019-124490
Published: 2019
Journal Articles

Adenylate Kinase 2 Links Energy Metabolism and Cell Fate in Hematopoietic Stem and Progenitor Cells

Free
Wenqing Wang, PhD, Avni Awani, PhD, Andrew Devilbiss, PhD, Thomas Mathews, PhD, Daniel Thomas, MD PhD, Daniel P. Dever, PhD, Matthew Porteus, MD PhD, Sean Morrison, PhD, Katja G Weinacht, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3705.
https://doi.org/10.1182/blood-2019-129424
Published: 2019
Journal Articles

Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing

Free
Selami Demirci, PhD, Jing Zeng, MD, Yuxuan Wu, PhD, Naoya Uchida, MD, Jackson Gamer, Morgan Yapundich, Claire Drysdale, Aylin C. Bonifacino, Allen E. Krouse, Nathaniel S. Linde, Robert E. Donahue, VMD, Anne H. Shen, Juan J. Haro-Mora, PhD, Alexis Leonard, MD, Tina Nassehi, Kevin Luk, Scot A. Wolfe, Cicera R. Lazzarotto, Shengdar Q. Tsai, PhD, Mitchell J. Weiss, MD, PhD, Daniel E. Bauer, MD PhD, John F. Tisdale, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4632.
https://doi.org/10.1182/blood-2019-129394
Published: 2019