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Gata2 Expression and Function in PML-RARA- driven Preleukemia and APL

Casey D.S. Katerndahl, PhD, Timothy J Ley, MD, Michelle A Cai, Timothy P Rooney, Jr., Sai Mukund Ramakrishnan, MS, Sridhar Nonavinkere Srivatsan, MS, Christopher A Miller, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 534.
https://doi.org/10.1182/blood-2019-131883
Published: 2019
Journal Articles

Preclinical Evaluation for Engraftment of Gene-Edited CD34+ Cells with a Sickle Cell Disease Mutation in a Rhesus Transplantation Model

Naoya Uchida, MD, Linhong Li, PhD, Tina Nassehi, Morgan Yapundich, Jackson Gamer, Claire Drysdale, Juan J. Haro-Mora, PhD, Selami Demirci, PhD, Aylin Bonifacino, MS, Allen Krouse, Nathaniel S. Linde, Cornell Allen, MS, Suk See De Ravin, MD, PhD, Robert E. Donahue, VMD, Harry L. Malech, MD, Madhusudan Peshwa, PhD, John F. Tisdale, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 609.
https://doi.org/10.1182/blood-2019-122727
Published: 2019
Journal Articles

Therapeutic Base Editing of Human Hematopoietic Stem Cells

Jing Zeng, MD, Yuxuan Wu, PhD, Chunyan Ren, PhD, Jasmine Bonanno, Anne H. Shen, Devlin Shea, Jason M Gehrke, PhD, John P Manis, MD, J. Keith Gehrke, Daniel E. Bauer, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 612.
https://doi.org/10.1182/blood-2019-129173
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

Regulation of Erythroblast Protein Expression By an RNA Splicing Mechanism, Decoy Exon-Mediated Intron Retention

Marilyn Parra, Weiguo Zhang, PhD, Jonathan Vu, Mark DeWitt, John G Conboy, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3504.
https://doi.org/10.1182/blood-2019-131153
Published: 2019
Journal Articles

Gene Editing and mRNA-Based Therapy: Two Complementary Therapeutic Approaches for the Treatment of Patients with Xmen Disease

Julie Brault, PhD PharmD, Ezekiel Bello, Ronald J. Meis, PhD, Taylor Liu, Sherry M. Koontz, BS, Kennichi Dowdell, PhD, Linhong Li, PhD, Cornell Allen, MS, Narda Theobald, BS, Janet Lee, BS, Aaron B. Clark, BS, Juan C. Ravell, MD, Michael Lenardo, MD, Gary A. Dahl, PhD, Harry L. Malech, MD, Suk See De Ravin, MD, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4637.
https://doi.org/10.1182/blood-2019-129062
Published: 2019
Journal Articles

Ex Vivo Expansion with E478 Increases the Rate of CRISPR-Mediated Homology Directed Repair of the Beta-Globin Gene in Human Hematopoietic Stem Cells By >10-Fold and Improves In Vivo Engraftment By >120-Fold

Kevin A. Goncalves, PhD, Megan D. Hoban, PhD, Sharon L. Hyzy, MS, Katia S. George, BA, Anthony E. Boitano, PhD, Michael P. Cooke, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4634.
https://doi.org/10.1182/blood-2019-131157
Published: 2019
Journal Articles

EDIT-301: An Experimental Autologous Cell Therapy Comprising Cas12a-RNP Modified mPB-CD34+ Cells for the Potential Treatment of SCD

Edouard De Dreuzy, PhD, Jack Heath, John A Zuris, PhD, Patricia Sousa, Ramya Viswanathan, Sean Scott, Jen Da Silva, Terence Ta, PhD, Stacy Capehart, PhD, Tongyao Wang, Cecilia Fernandez, PhD, Vic E Myer, PhD, Charles F Albright, PhD, Christopher J Wilson, PhD, Sandra Teixeira, PhD, Kai-Hsin Chang, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4636.
https://doi.org/10.1182/blood-2019-130256
Published: 2019
Journal Articles

Invariant NKT Cells Recognize Leukemia Cells with T-Cell and NK Receptors in a CD1d-Independent Manner

Takahiro Aoki, MD, Mariko Takami, Tomozumi Takatani, Kiwamu Motoyoshi, Ayana Ishii, Ayaka Hara, Takahide Toyoda, Reona Okada, Moeko Hino, MD, Ryo Koyama-Nasu, Masahiro Kiuchi, Kiyoshi Hirahara, Toshinori Nakayama, Naoki Shimojo, Shinichiro Motohashi
Journal: Blood
Blood (2019) 134 (Supplement_1): 3225.
https://doi.org/10.1182/blood-2019-121817
Published: 2019
Journal Articles

Donor-Derived CD19 CAR Cytokine Induced Killer (CIK) Cells Engineered with Sleeping Beauty Transposon for Relapsed B-Cell Acute Lymphoblastic Leukemia (B-ALL)

Chiara F Magnani, PhD, Giuseppe Gaipa, PhD, Daniela Belotti, Giada Matera, Sarah Tettamanti, PhD, Benedetta Cabiati, Chiara Buracchi, PhD, Grazia Fazio, Silvia Zaninelli, Silvia Rigamonti, Stefania Cesana, Valentina Colombo, Giovanni Cazzaniga, PhD, Attilio Rovelli, MD, Adriana Balduzzi, MD, Sara Napolitano, MD, Fabrizio Benedicenti, Eugenio Montini, PhD, Gian Maria Borleri, Silvia Ferrari, MD, Giuseppe Gritti, MD PhD, Federico Lussana, MD, Martino Introna, MD, Alessandro Rambaldi, MD, Giuseppe Dastoli, MD, Andrea Biondi, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 200.
https://doi.org/10.1182/blood-2019-125894
Published: 2019
Journal Articles

Manufacture of an Allogeneic CAR-T Stem Cell Memory Product Candidate for Multiple Myeloma, P-Bcma-ALLO1, Is Robust, Reproducible and Highly Scalable

Stacey A Cranert, PhD, Maximilian Richter, PhD, Min Tong, MS, Leslie Weiss, MS, Yening Tan, MS, Eric M. Ostertag, MD PhD, Julia Coronella, PhD, Devon J Shedlock, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4445.
https://doi.org/10.1182/blood-2019-131839
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

A Functional Analysis of SNP on Anthracycline-Induced Cardiotoxicity in a Uniform Genetic Background Using CRISPR/Cas9 Mediated Single-Base-Edited iPSCs

Takayuki Nakamura, MD, Shinichi Mizuno, MD PhD, Hidetoshi Ozawa, Yoshitaka Yamasaki, MD, Shuki Oya, MD, Satoshi Morishige, MD, Kazutoshi Aoyama, MD PhD, Maki Ymaguchi, MD PhD, Ritsuko Seki, PhD, Fumihiko Mouri, MD PhD, Koichi Osaki, MD, Koji Nagafuji, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 5748.
https://doi.org/10.1182/blood-2019-123932
Published: 2019
Journal Articles

Rapid Personalized Manufacture (RPM) of Sleeping Beauty System-Generated NY-ESO-1-Specific TCR-T Cells Co-Expressing Membrane-Bound IL-15 Yields Anti-Tumor Responses

Lenka V Hurton, PhD, Tiejuan Mi, Kirsten C Switzer, PhD, Ling Zhang, Cuiping Dai, Misun Kim, Karishma R Bavisi, Matthew J Figliola, Vazquez M Lydia, Sourindra N Maiti, Shihuang Su, Drew C Deniger, PhD, Simon Olivares, Laurence J N Cooper, MD PhD, Harjeet Singh, MSc, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3218.
https://doi.org/10.1182/blood-2019-131419
Published: 2019
Journal Articles

Universal' Fratricide-Resistant CAR T Cells Against T Cell Leukemia Generated By Coupled & Uncoupled Deamination Mediated Base Editing

Christos Georgiadis, PhD, Jane Rasaiyaah, PhD, Soragia Athina Gkazi, PhD, Roland Preece, Annie Etuk, PhD, Abraham Christi, Waseem Qasim, MBBS, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3219.
https://doi.org/10.1182/blood-2019-130057
Published: 2019
Journal Articles

Targeting CD38 high Acute Myeloid Leukaemia with "Affinity Optimized" Chimeric Antigen Receptor and Membrane Bound TRAIL Expressing Natural Killer Cells

Emma Nolan, BSc, Arwen Stikvoort, PhD, Mark Gurney, MD, Nutsa Burduli, BSc, Lucy Kirkham-McCarthy, MSc, John Daly, MSc, Niels W. C. J. Van De Donk, Tuna Mutis, MD PhD, Subhashis Sarkar, PhD, Michael E O'Dwyer, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 5536.
https://doi.org/10.1182/blood-2019-128605
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

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

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

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