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Journal Articles

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

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

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)

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

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

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
Journal Articles

Infection Is a Driver of Dnmt3a-Mutant Clonal Hematopoiesis

Daniel Hormaechea Agulla, PhD, Katie A Matatall, PhD, Duy Le, PhDcandidate, Bailee Nicole Kain, PhDcandidate, Roman Jaksik, PhD, Marek Kimmel, PhD DSc, Grant Challen, PhD, Katherine Y. King, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 817.
https://doi.org/10.1182/blood-2019-126831
Published: 2019
Journal Articles

Manufacturing Scale-up and Preclinical Development of ET-01, Autologous CD34 + Cells with the BCL11A Erythroid Enhancer Edited By CRISPR/Cas9, for Patients with β-Thalassemia Major

Riguo Fang, PhD, Pengfei Yuan, PhD, Lingling Yu, Bachelor, Huihui Yang, Master, Jie Liu, Master, Jia Shi, PhD, Yongjian Zhang, PhD, Wei Zhang, PhD, Yiding Zhao, PhD, Zhiyong Peng, MD, Chunfu Li, MD, Yun Li, MD, Dong Wei, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 965.
https://doi.org/10.1182/blood-2019-126499
Published: 2019
Journal Articles

CD162 Is a Key E-Selectin Receptor Promoting Acute Myeloid Leukemia Chemo-Resistance in the Bone Marrow Niche

Johanna D Erbani, Joshua Tay, PhD, Valerie Barbier, Masters, Jean-Pierre Levesque, PhD, John L. Magnani, PhD, Ingrid G Winkler, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 907.
https://doi.org/10.1182/blood-2019-132233
Published: 2019
Journal Articles

Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer Results in Enriched Biallelic Editing, Increased Fetal Hemoglobin, and Reduced Sickling in Erythroid Cells Derived from Sickle Cell Disease Patients

Samuel Lessard, PhD, Pauline Rimmele, PhD, Hui Ling, PhD, Kevin Moran, MSc, Benjamin Vieira, BS, Yi-Dong Lin, PhD, Vu Hong, PhD, Andreas Reik, PhD, Denny Dang, Naoya Uchida, MD, John F. Tisdale, MD, Pablo Rendo, MD, Ahmed Daak, PhD, Alexandra Hicks, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 974.
https://doi.org/10.1182/blood-2019-126048
Published: 2019
Journal Articles

Chromosomal Loop Editing Reveals New Epigenomic Landscape in the β-Globin Locus with Implications for Hemoglobinopathy Therapies

Pamela Himadewi, BSc, Xiaotian Zhang, PhDBS, Haley Gore, MS, Xue Qing David Wang, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 813.
https://doi.org/10.1182/blood-2019-125020
Published: 2019
Journal Articles

Venetoclax Resistance in Mantle Cell Lymphoma Is Mediated By BCL-XL and Can be Circumvent By Inhibiting the BH4 Domain of BCL-2

Daniela Steinbrecher, PhD, Felix Seyfried, MD, Eugen Tausch, Johannes Bloehdorn, MD, Billy Michael Chelliah Jebaraj, PhD, Klaus-Michael Debatin, MD, Lüder Hinrich Meyer, MD, Hartmut Döhner, MD, Stephan Stilgenbauer, MD, Christof Schneider, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1507.
https://doi.org/10.1182/blood-2019-127931
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)

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

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

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

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

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

Gene Editing ELANE in Human Hematopoietic Stem and Progenitor Cells Reveals Disease Mechanisms and Therapeutic Strategies for Severe Congenital Neutropenia

Shuquan Rao, PhD, Josias Brito-Frazao, Anna V. Serbin, Qiuming Yao, PhD, Kevin Luk, Yuxuan Wu, PhD, Jing Zeng, MD, Chunyan Ren, PhD, Ruth Watkinson, Myriam Armant, PhD, Luca Pinello, PhD, Akiko Shimamura, MD PhD, Benhur Lee, Scot A. Wolfe, PhD, Peter E. Newburger, MD, Daniel E. Bauer, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3.
https://doi.org/10.1182/blood-2019-131073
Published: 2019
Journal Articles

First-in-Human Assessment of Feasibility and Safety of Multiplexed Genetic Engineering of Autologous T Cells Expressing NY-ESO -1 TCR and CRISPR/Cas9 Gene Edited to Eliminate Endogenous TCR and PD-1 (NYCE T cells) in Advanced Multiple Myeloma (MM) and Sarcoma

Edward A. Stadtmauer, MD FACP, Adam D. Cohen, MD, Kristy Weber, MD, Simon F Lacey, PhD, Vanessa E. Gonzalez, J. Joseph Melenhorst, PhD, Joseph A Fraietta, PhD, Gabriela Plesa, MD, Joanne Shea, Tina Matlawski, RN, Amanda Cervini, RN, Patricia Mangan, CRNP, Avery Gaymon, Stephanie Desjardins, Eric Lancaster, MD, January Salas-Mckee, Megan M. Suhoski, PhD, Andrew Fesnak, MD, Matthew O'Rourke, Anne Lamontagne, Don L. Siegel, MD PhD, Regina M Young, PhD, Anne Chew, PhD, Christopher L Nobles, PhD, Frederic D. Bushman, PhD, Howard Y. Chang, MD PhD, Ansuman T. Satpathy, MD PhD, Yangbing Zhao, PhD MD, Wei-Ting Hwang, PhD, Elizabeth O. Hexner, MDMS, Carl H June, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 49.
https://doi.org/10.1182/blood-2019-122374
Published: 2019
Journal Articles

Next Generation CRISPR Gene-Edited and Off-the-Shelf Virus-Specific T-Cells for the Immunocompromised Patient

Rafet Basar, MD, May Daher, MD, Nadima Uprety, MS, Elif Gokdemir, BS, Abdullah Saleh Alsuliman, PhD, Mayela Carolina Mendt, PhD, Lucila Kerbauy, MD, Mayra Shanley, PhD, Gonca Ozcan, MD, Emily Ensley, BS, Ana Karen Nunez Cortes, MD, Li Li, MD, Junjun Lu, Mecit Kaplan, MS, Vandana Nandivada, BS, MS, Mustafa Bdaiwi, MD, Ye Li, Luciana Melo Garcia, MD, Enli Liu, MD, Sonny Ang, PhD, Pinaki P Banerjee, PhD, Richard E. Champlin, MD, Elizabeth J. Shpall, MD, Katayoun Rezvani, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1944.
https://doi.org/10.1182/blood-2019-125276
Published: 2019
Journal Articles

CRISPR-Cas9 Genome Editing of γ-Globin Promoters in Human Hematopoietic Stem Cells to Induce Erythrocyte Fetal Hemoglobin for Treatment of β-Hemoglobinopathies

Jean-Yves Metais, PhD, Phillip A Doerfler, PhD, Thiyagaraj Mayuranathan, Daniel E. Bauer, MD PhD, Stephanie Fowler, Matthew Hsieh, MD, Varun Katta, Sagar Keriwala, Cicera Lazzarotto, Kevin Luk, Michael Neel, Scott Perry, Shaina Porter, Byoung Y. Ryu, PhD, Akshay Sharma, MBBS, Devlin Shea, Samuel Peters, John F. Tisdale, MD, Scot A. Wolfe, PhD, Kaitly Jensen Woodard, BS, Naoya Uchida, MD, Yuxuan Wu, PhD, Yu Yao, MD, Jing Zeng, MD, Shondra M. Pruett-Miller, PhD, Shengdar Q. Tsai, PhD, Mitchell J. Weiss, MD, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2066.
https://doi.org/10.1182/blood-2019-131318
Published: 2019
Journal Articles

Enabling Gene-Edited, Regulatory-like, T Cells (edTreg) for Treatment of IPEX and Other Autoimmune Disorders

Yuchi Honaker, PhD, Karen Sommer, PhD, Noelle Dahl, Yufei Xiang, MD, PhD, Christina Lopez, PhD, Samuel Scharffenberger, Michelle Christian, Troy R. Torgerson, MD PhD, Andrew M. Scharenberg, MD, David J. Rawlings, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 2071.
https://doi.org/10.1182/blood-2019-131946
Published: 2019
Journal Articles

Germline Mutations Predispose to Familial Myeloproliferative Neoplasms

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