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Journal Articles

Efficient and Specific Multi-Locus Editing of Allogeneic CAR T Cells for Hypoimmunity during Large Scale Manufacture Using Cas12b

Blood (2022) 140 (Supplement 1): 1606–1607.

https://doi.org/10.1182/blood-2022-167627

Published: 2022

Journal Articles

Engineered Hypoimmune Allogeneic CAR T Cells Exhibit Innate and Adaptive Immune Evasion Even after Sensitization in Humanized Mice and Retain Potent Anti-Tumor Activity

Xiaomeng Hu, Mo Dao, Kathy White, Corie Gattis, Ryan Clarke, Sam Landry, Ron Basco, Eleonore Tham, Andrew Tucker, Emily Luo, Christopher Bandoro, Elaine Chu, Chi Young, Aaron E. Foster, William E Dowdle, Edward J Rebar, Terry J. Fry, Sonja Schrepfer

Blood (2021) 138 (Supplement 1): 1690.

https://doi.org/10.1182/blood-2021-150021

Published: 2021

Journal Articles

A Potential Therapy for Beta-Thalassemia (ST-400) and Sickle Cell Disease (BIVV003)

Michael C. Holmes, PhD, Andreas Reik, PhD, Edward J. Rebar, PhD BS, Jeffrey C. Miller, PhD, Yuanyue Zhou, Lei Zhang, PhD, Patrick Li, Sagar Vaidya

Blood (2017) 130 (Supplement 1): 2066.

https://doi.org/10.1182/blood.V130.Suppl_1.2066.2066

Published: 2017

Journal Articles

Long-term multilineage engraftment of autologous genome-edited hematopoietic stem cells in nonhuman primates

Christopher W. Peterson, Jianbin Wang, Krystin K. Norman, Zachary K. Norgaard, Olivier Humbert, Collette K. Tse, Jenny J. Yan, Richard G. Trimble, David A. Shivak, Edward J. Rebar, Philip D. Gregory, Michael C. Holmes, Hans-Peter Kiem

Blood (2016) 127 (20): 2416–2426.

https://doi.org/10.1182/blood-2015-09-672337

Published: 2016

Includes: Supplemental data

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

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

ZFN-Mediated Gene Targeting at the Albumin Locus in Liver Results in Therapeutic Levels of Human FIX in Mice and Non-Human Primates

Thomas Wechsler, PhD, Kathleen E. Meyer, S. Kaye Spratt, Judith Greengard, PhD, Yolanda Santiago, Scott Sproul, Richard Surosky, David E. Paschon, Nathalie Dubois-Stringfellow, Dale Genji Ando, MD, Geoff Nichol, Edward J. Rebar, PhD, Michael C. Holmes, PhD

Blood (2015) 126 (23): 200.

https://doi.org/10.1182/blood.V126.23.200.200

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

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

Rajiv Sharma, Xavier M. Anguela, Yannick Doyon, Thomas Wechsler, Russell C. DeKelver, Scott Sproul, David E. Paschon, Jeffrey C. Miller, Robert J. Davidson, David Shivak, Shangzhen Zhou, Julianne Rieders, Philip D. Gregory, Michael C. Holmes, Edward J. Rebar, Katherine A. High

Blood (2015) 126 (15): 1777–1784.

https://doi.org/10.1182/blood-2014-12-615492

Published: 2015

Includes: Supplemental data

Journal Articles

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

Blood (2015) 125 (17): 2597–2604.

https://doi.org/10.1182/blood-2014-12-615948

Published: 2015

Includes: Supplemental data

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

ZFN Mediated Targeting Of Albumin “Safe Harbor” Results In Therapeutic Levels Of Human Factor VIII In a Mouse Model Of Hemophilia A

Xavier M Anguela, PhD, Rajiv Sharma, Yannick Doyon, PhD, Thomas Wechsler, PhD, David E Paschon, PhD, Robert J Davidson, Shangzhen Zhou, MD, Philip D Gregory, PhD, Michael C. Holmes, PhD, Edward J Rebar, PhD, Katherine A. High, MD

Blood (2013) 122 (21): 720.

https://doi.org/10.1182/blood.V122.21.720.720

Published: 2013

Journal Articles

Toward eliminating HLA class I expression to generate universal cells from allogeneic donors

Blood (2013) 122 (8): 1341–1349.

https://doi.org/10.1182/blood-2013-03-478255

Published: 2013

Includes: Supplemental data

Journal Articles

A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR

Hiroki Torikai, Andreas Reik, Pei-Qi Liu, Yuanyue Zhou, Ling Zhang, Sourindra Maiti, Helen Huls, Jeffrey C. Miller, Partow Kebriaei, Brian Rabinovitch, Dean A. Lee, Richard E. Champlin, Chiara Bonini, Luigi Naldini, Edward J. Rebar, Philip D. Gregory, Michael C. Holmes, Laurence J. N. Cooper

Blood (2012) 119 (24): 5697–5705.

https://doi.org/10.1182/blood-2012-01-405365

Published: 2012

Includes: Supplemental data

Journal Articles

HLA and TCR Knockout by Zinc Finger Nucleases: Toward “off-the-Shelf” Allogeneic T-Cell Therapy for CD19 + Malignancies.

Hiroki Torikai, Andreas Reik, Carrie Yuen, Yuanyue Zhou, Denise Kellar, Helen Huls, Edus H. Warren, III, Scott S. Tykodi, Philip D. Gregory, Michael C. Holmes, Edward J. Rebar, Dean A. Lee, Richard E. Champlin, Chiara Bonini, Laurence Cooper

Blood (2010) 116 (21): 3766.

https://doi.org/10.1182/blood.V116.21.3766.3766

Published: 2010

Journal Articles

“Designer” Cells for Cellular Immunotherapy: Zinc Finger Nuclease (ZFN) Stimulated Targeted Recombination for the Simultaneous Disruption of the Endogenous Glucocorticoid Receptor and Site-Specific Addition of the IL13-Zetakine.

Andreas Reik, PhD, Michael C. Holmes, Ph.D., Fyodor D. Urnov, Ph.D., Yuanyue Zhou, Matthew Mendel, Pei-Qi Liu, Ph.D., Erica Moehle, Edward J. Rebar, Ph.D., Jeffrey C. Miller, Ph.D., Dale Ando, M.D., Philip D. Gregory, Ph.D., Michael Jensen, M.D.

Blood (2006) 108 (11): 3705.

https://doi.org/10.1182/blood.V108.11.3705.3705

Published: 2006

NARROW

Efficient and Specific Multi-Locus Editing of Allogeneic CAR T Cells for Hypoimmunity during Large Scale Manufacture Using Cas12b

Engineered Hypoimmune Allogeneic CAR T Cells Exhibit Innate and Adaptive Immune Evasion Even after Sensitization in Humanized Mice and Retain Potent Anti-Tumor Activity

A Potential Therapy for Beta-Thalassemia (ST-400) and Sickle Cell Disease (BIVV003)

Long-term multilineage engraftment of autologous genome-edited hematopoietic stem cells in nonhuman primates

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

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

ZFN-Mediated Gene Targeting at the Albumin Locus in Liver Results in Therapeutic Levels of Human FIX in Mice and Non-Human Primates

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

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

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

ZFN Mediated Targeting Of Albumin “Safe Harbor” Results In Therapeutic Levels Of Human Factor VIII In a Mouse Model Of Hemophilia A

Toward eliminating HLA class I expression to generate universal cells from allogeneic donors

A foundation for universal T-cell based immunotherapy: T cells engineered to express a CD19-specific chimeric-antigen-receptor and eliminate expression of endogenous TCR

HLA and TCR Knockout by Zinc Finger Nucleases: Toward “off-the-Shelf” Allogeneic T-Cell Therapy for CD19 + Malignancies.

“Designer” Cells for Cellular Immunotherapy: Zinc Finger Nuclease (ZFN) Stimulated Targeted Recombination for the Simultaneous Disruption of the Endogenous Glucocorticoid Receptor and Site-Specific Addition of the IL13-Zetakine.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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