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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

Blood (2019) 134 (Supplement_1): 965.

https://doi.org/10.1182/blood-2019-126499

Published: 2019

Journal Articles

Notch1 mutation in B Chronic Lymphocytic Lymphocytes Induces Proliferation through Cell Cycle G1/S Phase-Specific Cyclin Overexpression

Ana M Hurtado López, Tzu Hua Chen-Liang, María Zurdo, Anna Puiggros, PhD, Andrea Gómez-Llonín, B.Sc., María-Dolores García-Malo, MD, Francisco José Ortuño, Vicente Vicente Garcia, MD PhD, Blanca Espinet, PhD, Andres Jerez

Blood (2019) 134 (Supplement_1): 851.

https://doi.org/10.1182/blood-2019-127960

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

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

Blood (2019) 134 (Supplement_1): 612.

https://doi.org/10.1182/blood-2019-129173

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

Blood (2019) 134 (Supplement_1): 5536.

https://doi.org/10.1182/blood-2019-128605

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

Blood (2019) 134 (Supplement_1): 5754.

https://doi.org/10.1182/blood-2019-129931

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

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

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

Blood (2019) 134 (Supplement_1): 2066.

https://doi.org/10.1182/blood-2019-131318

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

Blood (2019) 134 (Supplement_1): 3225.

https://doi.org/10.1182/blood-2019-121817

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

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

Blood (2019) 134 (Supplement_1): 3237.

https://doi.org/10.1182/blood-2019-129162

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

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

Blood (2019) 134 (Supplement_1): 3219.

https://doi.org/10.1182/blood-2019-130057

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

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

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

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

Blood (2019) 134 (Supplement_1): 4636.

https://doi.org/10.1182/blood-2019-130256

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

Blood (2019) 134 (Supplement_1): 4445.

https://doi.org/10.1182/blood-2019-131839

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

Blood (2019) 134 (Supplement_1): 5748.

https://doi.org/10.1182/blood-2019-123932

Published: 2019

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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

Notch1 mutation in B Chronic Lymphocytic Lymphocytes Induces Proliferation through Cell Cycle G1/S Phase-Specific Cyclin Overexpression

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

Therapeutic Base Editing of Human Hematopoietic Stem Cells

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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