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Journal Articles

Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Successful Engraftment, Increased Hemoglobin, and Reduced Transfusion Dependence in Patients with Transfusion-Dependent Beta-Thalassemia Treated in the Edithal Trial

Haydar Frangoul, Rabi Hanna, Mark C. Walters, Kai-Hsin Chang, Michael Jaskolka, Keunpyo Kim, Baisong Mei, Olubunmi Afonja, Alexis Thompson

Blood (2024) 144 (Supplement 1): 7456.

https://doi.org/10.1182/blood-2024-208623

Published: 2024

Journal Articles

Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Sustained Hemoglobin Normalization and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease Treated in the RUBY Trial

Rabi Hanna, Haydar Frangoul, Christopher McKinney, Luis Pineiro, Markus Mapara, Jignesh Dalal, Hemalatha Rangarajan, Harold Atkins, Kai-Hsin Chang, Baisong Mei, Olubunmi Afonja, Mark C. Walters

Blood (2024) 144 (Supplement 1): 4955.

https://doi.org/10.1182/blood-2024-210343

Published: 2024

Journal Articles

AsCas12a Gene Editing of HBG1/2 Promoters with EDIT-301 Results in Rapid and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia

Rabi Hanna, Haydar Frangoul, Christopher McKinney, Luis Pineiro, Markus Mapara, Jignesh Dalal, Kai-Hsin Chang, Michael Jaskolka, Keunpyo Kim, Daphne L. Farrington, Maha Wally, Baisong Mei, Adebayo Lawal, Olubunmi O. Afonja, Mark C. Walters

Blood (2023) 142 (Supplement 1): 4996.

https://doi.org/10.1182/blood-2023-187397

Published: 2023

Journal Articles

Deletion of CISH and TGFβR2 in iPSC-Derived NK Cells Promotes High Cytotoxicity and Enhances In Vivo Tumor Killing

Alexandra Gerew, Steven Sexton, Kevin M Wasko, Mark S Shearman, Kate Zhang, Kai-Hsin Chang, Samia Q. Khan

Blood (2021) 138 (Supplement 1): 2780.

https://doi.org/10.1182/blood-2021-150731

Published: 2021

Journal Articles

Preclinical Development of EDIT301, an Autologous Cell Therapy Comprising AsCas12a-RNP Modified Mobilized Peripheral Blood-CD34 + Cells for the Potential Treatment of Transfusion Dependent Beta Thalassemia

Patricia Sousa, Tusneem Janoudi, Edouard deDreuzy, Mark S Shearman, Kate Zhang, Kai-Hsin Chang

Blood (2021) 138 (Supplement 1): 1858.

https://doi.org/10.1182/blood-2021-149956

Published: 2021

Journal Articles

A Bicistronic Vector Expressing CD16 and a Membrane Bound IL-15 Construct in iPSC Derived NK Cells Increased Cytotoxicity and Persistence

Alexander G Allen, Rithu Pattali, Kaitlyn M Izzo, Jared A Getgano, Kevin M Wasko, Laura C Blaha, John A Zuris, Kate Zhang, Mark S Shearman, Kai-Hsin Chang

Blood (2021) 138 (Supplement 1): 4809.

https://doi.org/10.1182/blood-2021-153258

Published: 2021

Journal Articles

Robust Pre-Clinical Results and Large-Scale Manufacturing Process for Edit-301: An Autologous Cell Therapy for the Potential Treatment of SCD

Edouard De Dreuzy, PhD, Jack Heath, Patricia Sousa, Tusneem Janoudi, Harry An, PhD, Scott Hansen, David K. Wood, PhD, Charles F Albright, PhD, Sandra Teixeira, PhD, Tamara Monesmith, Kate Zhang, PhD, Kai-Hsin Chang, PhD

Blood (2020) 136 (Supplement 1): 45–46.

https://doi.org/10.1182/blood-2020-140073

Published: 2020

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

Comparative Studies Reveal Robust HbF Induction By Editing of HBG1/2 Promoters or BCL11A Erythroid-Enhancer in Human CD34+ Cells but That BCL11A Erythroid-Enhancer Editing Is Associated with Selective Reduction in Erythroid Lineage Reconstitution in a Xenotransplantation Model

Blood (2018) 132 (Supplement 1): 409.

https://doi.org/10.1182/blood-2018-99-114780

Published: 2018

Journal Articles

Genome Editing of the Bcl11A Erythroid Specific Enhancer in Bone Marrow Derived Hematopoietic Stem and Progenitor Cells for the Treatment of Sickle Cell Disease

Siyuan Tan, PhD, Kai-Hsin Chang, PhD, Sarah Smith, BS, Kai Chen, MS, Timonthy Sullivan, BS, Qianhe Zhou, PhD, Andreas Reik, PhD, Fyodor D Urnov, PhD, Edward J. Rebar, PhD, Olivier Danos, PhD, Haiyan Jiang, PhD

Blood (2015) 126 (23): 203.

https://doi.org/10.1182/blood.V126.23.203.203

Published: 2015

Journal Articles

Clonal Analysis of Human Bone Marrow CD34+ Cells Edited By BCL11A-Targeting Zinc Finger Nucleases Reveals Clinically Relevant Levels of Fetal Globin Expression in Edited Erythroid Progeny

Kai-Hsin Chang, PhD, Timothy Sullivan, MS, Mei Liu, BS, Xiao Yang, PhD, Chao Sun, PhD, Benjamin Vieira, BS, Ming Zhang, PhD, Vu P. Hong, PhD, Kai Chen, MS, Sarah Smith, BS, Siyuan Tan, PhD, Andreas Reik, PhD, Fyodor D Urnov, PhD, Edward J. Rebar, PhD, Olivier Danos, PhD, Haiyan Jiang, PhD

Blood (2015) 126 (23): 3234.

https://doi.org/10.1182/blood.V126.23.3234.3234

Published: 2015

Journal Articles

Clinical-Scale Genome Editing of the Human BCL11A Erythroid Enhancer for Treatment of the Hemoglobinopathies

Fyodor D Urnov, PhD, Andreas Reik, PhD, Jeff Vierstra, PhD, Kai-Hsin Chang, PhD, Yuanyue Zhou, Andrea Mich, Jennifer Adrian, Lisa Fox, Nikoletta Psatha, PhD, Siyuan Tan, PhD, Evangelia Yannaki, MD, Stewart Craig, PhD, Edward J. Rebar, PhD, John Stamatoyannopoulos, MD, Thalia Papayannopoulou, MD, George Stamatoyannopoulos, MD DSci, Haiyan Jiang, PhD

Blood (2015) 126 (23): 204.

https://doi.org/10.1182/blood.V126.23.204.204

Published: 2015

Journal Articles

Targeted Gene Modification In Hematopoietic Stem Cells: A Potential Treatment For Thalassemia and Sickle Cell Anemia

Blood (2013) 122 (21): 434.

https://doi.org/10.1182/blood.V122.21.434.434

Published: 2013

Journal Articles

Deciphering the Cis- and Trans- regulatory Roles of KLF6 in Primitive Hematopoiesis

Qian Xiong, Zhaojun Zhang, Hongzhu Qu, Xiuyan Ruan, Hai Wang, Qian Zhang, Heyuan Qi, Yanming Li, Hongying Sun, Kai-Hsin Chang, George Stamatoyannopoulos, John A. Stamatoyannopoulos, MD, Xiangdong Fang

Blood (2012) 120 (21): 4730.

https://doi.org/10.1182/blood.V120.21.4730.4730

Published: 2012

Journal Articles

Comparative Blood Group Profiling of Human Erythroid Cells (EBs) Generated from Adult Blood (AB), Cord Blood (CB), Human Embryonic Stem Cells (hESC) and Induced Pluripotent Stem Cells (iPS)

Barbara Ghinassi, Maria Themeli, MD, Kai-Hsin Chang, Gregory Halverson, Ghazala Hashmi, Michel Sadelain, MD, PhD, Thalia Papayannopoulou, Anna Rita F Migliaccio, PhD, Carolyn Whitsett

Blood (2011) 118 (21): 1027.

https://doi.org/10.1182/blood.V118.21.1027.1027

Published: 2011

Journal Articles

Globin phenotype of erythroid cells derived from human induced pluripotent stem cells

Kai-Hsin Chang, Andy Huang, Roli K. Hirata, Pei-Rong Wang, David W. Russell, Thalia Papayannopoulou

Blood (2010) 115 (12): 2553–2554.

https://doi.org/10.1182/blood-2009-11-252650

Published: 2010

Journal Articles

Chromatin Profiling of the Globin Loci of Human ES Cells and ES-Derived Erythroid Cells.

Xiangdong Fang, PhD, MD, Kai-Hsin Chang, Daniel Bates, Morgan Diegel, Richard Sandstrom, Molly Weaver, Michael O. Dorschner, Peter J. Sabo, Thalia Papayannopoulou, MD, George Stamatoyannopoulos, MD, DSci, John Stamatoyannopoulos

Blood (2009) 114 (22): 2535.

https://doi.org/10.1182/blood.V114.22.2535.2535

Published: 2009

Journal Articles

Increased numbers of circulating hematopoietic stem/progenitor cells are chronically maintained in patients treated with the CD49d blocking antibody natalizumab

Halvard Bonig, Annette Wundes, Kai-Hsin Chang, Sylvia Lucas, Thalia Papayannopoulou

Blood (2008) 111 (7): 3439–3441.

https://doi.org/10.1182/blood-2007-09-112052

Published: 2008

Journal Articles

Hematopoietic Stem/Progenitor Cells (HSPC) Mobilization Parameters in Patients Chronically Treated with the CD49d Blocking Antibody Natalizumab.

Halvard Bonig, MD, Annette Wundes, M.D., Kai-Hsin Chang, Ph.D., Sylvia Lucas, M.D., Ph.D., Thalia Papayannopoulou, M.D.

Blood (2007) 110 (11): 177.

https://doi.org/10.1182/blood.V110.11.177.177

Published: 2007

Journal Articles

Definitive-like erythroid cells derived from human embryonic stem cells coexpress high levels of embryonic and fetal globins with little or no adult globin

Kai-Hsin Chang, Angelique M. Nelson, Hua Cao, Linlin Wang, Betty Nakamoto, Carol B. Ware, Thalia Papayannopoulou

Blood (2006) 108 (5): 1515–1523.

https://doi.org/10.1182/blood-2005-11-011874

Published: 2006

NARROW

Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Successful Engraftment, Increased Hemoglobin, and Reduced Transfusion Dependence in Patients with Transfusion-Dependent Beta-Thalassemia Treated in the Edithal Trial

Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Sustained Hemoglobin Normalization and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease Treated in the RUBY Trial

AsCas12a Gene Editing of HBG1/2 Promoters with EDIT-301 Results in Rapid and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia

Deletion of CISH and TGFβR2 in iPSC-Derived NK Cells Promotes High Cytotoxicity and Enhances In Vivo Tumor Killing

Preclinical Development of EDIT301, an Autologous Cell Therapy Comprising AsCas12a-RNP Modified Mobilized Peripheral Blood-CD34 + Cells for the Potential Treatment of Transfusion Dependent Beta Thalassemia

A Bicistronic Vector Expressing CD16 and a Membrane Bound IL-15 Construct in iPSC Derived NK Cells Increased Cytotoxicity and Persistence

Robust Pre-Clinical Results and Large-Scale Manufacturing Process for Edit-301: An Autologous Cell Therapy for the Potential Treatment of SCD

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

Comparative Studies Reveal Robust HbF Induction By Editing of HBG1/2 Promoters or BCL11A Erythroid-Enhancer in Human CD34+ Cells but That BCL11A Erythroid-Enhancer Editing Is Associated with Selective Reduction in Erythroid Lineage Reconstitution in a Xenotransplantation Model

Genome Editing of the Bcl11A Erythroid Specific Enhancer in Bone Marrow Derived Hematopoietic Stem and Progenitor Cells for the Treatment of Sickle Cell Disease

Clonal Analysis of Human Bone Marrow CD34+ Cells Edited By BCL11A-Targeting Zinc Finger Nucleases Reveals Clinically Relevant Levels of Fetal Globin Expression in Edited Erythroid Progeny

Clinical-Scale Genome Editing of the Human BCL11A Erythroid Enhancer for Treatment of the Hemoglobinopathies

Targeted Gene Modification In Hematopoietic Stem Cells: A Potential Treatment For Thalassemia and Sickle Cell Anemia

Deciphering the Cis- and Trans- regulatory Roles of KLF6 in Primitive Hematopoiesis

Comparative Blood Group Profiling of Human Erythroid Cells (EBs) Generated from Adult Blood (AB), Cord Blood (CB), Human Embryonic Stem Cells (hESC) and Induced Pluripotent Stem Cells (iPS)

Globin phenotype of erythroid cells derived from human induced pluripotent stem cells

Chromatin Profiling of the Globin Loci of Human ES Cells and ES-Derived Erythroid Cells.

Increased numbers of circulating hematopoietic stem/progenitor cells are chronically maintained in patients treated with the CD49d blocking antibody natalizumab

Hematopoietic Stem/Progenitor Cells (HSPC) Mobilization Parameters in Patients Chronically Treated with the CD49d Blocking Antibody Natalizumab.

Definitive-like erythroid cells derived from human embryonic stem cells coexpress high levels of embryonic and fetal globins with little or no adult globin

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American Society of Hematology

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American Society of Hematology

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