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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

Blood (2019) 134 (Supplement_1): 974.

https://doi.org/10.1182/blood-2019-126048

Published: 2019

Journal Articles

Ex Vivo Gene-Edited Cell Therapy for Sickle Cell Disease: Disruption of the BCL11A Erythroid Enhancer with Zinc Finger Nucleases Increases Fetal Hemoglobin in Plerixafor Mobilized Human CD34+ Cells

Kevin Moran, MSc, Hui Ling, PhD, Samuel Lessard, PhD, Benjamin Vieira, Vu Hong, PhD, Michael C. Holmes, PhD, Andreas Reik, PhD, Denny Dang, David Gray, Dana Levasseur, PhD, Pauline Rimmele, PhD

Blood (2018) 132 (Supplement 1): 2190.

https://doi.org/10.1182/blood-2018-99-116998

Published: 2018

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

NY-ESO-1 TCR single edited stem and central memory T cells to treat multiple myeloma without graft-versus-host disease

Blood (2017) 130 (5): 606–618.

https://doi.org/10.1182/blood-2016-08-732636

Published: 2017

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

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

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

NY-ESO-1 Single Edited T Cells to Treat Multiple Myeloma without Inducing GvHD

Sara Mastaglio, MD, Pietro Genovese, PhD, Zulma Magnani, MSc, Elisa Landoni, Barbara Camisa, BSc, Giulia Schiroli, Elena Provasi, PhD, Angelo Lombardo, PhD, Andreas Reik, PhD, Nicoletta Cieri, MD PhD, Maurilio Ponzoni, Fabio Ciceri, MD, Claudio Bordignon, MD, Michael C. Holmes, PhD, Philip D Gregory, PhD, Luigi Naldini, MD PhD, Chiara Bonini, MD

Blood (2014) 124 (21): 308.

https://doi.org/10.1182/blood.V124.21.308.308

Published: 2014

Journal Articles

TCR Gene Editing Achieved In a Single Round Of T Cell Activation Is Sufficient To Redirect T Cell Specificity and Prevent GvHD

Sara Mastaglio, MD, Pietro Genovese, PhD, Zulma Magnani, MSc, Elena Provasi, PhD, Angelo Lombardo, PhD, Andreas Reik, Nicoletta Cieri, MD, Fabio Ciceri, Claudio Bordignon, MD, Michael C. Holmes, PhD, Philip D Gregory, PhD, Luigi Naldini, MD, PhD, Chiara Bonini, MD

Blood (2013) 122 (21): 2898.

https://doi.org/10.1182/blood.V122.21.2898.2898

Published: 2013

Journal Articles

Zinc Finger Nucleases Targeting The β-Globin Locus Drive Efficient Correction Of The Sickle Mutation In CD34 + Cells

Megan D Hoban, BS, Alok V Joglekar, PhD, David Gray, BS, Michael L Kaufman, MS, Fabrizia Urbinati, PhD, Shantha Senadheera, MS, Gregory Cost, PhD, Andreas Reik, PhD, Michael C. Holmes, PhD, Philip D Gregory, PhD, Roger P. Hollis, PhD, Donald B. Kohn, MD

Blood (2013) 122 (21): 2904.

https://doi.org/10.1182/blood.V122.21.2904.2904

Published: 2013

Journal Articles

Autologous Hematopoietic Stem/Progenitor Cell (HSPC) Therapy For Monogenic Blood Disorders: Scalable, cGMP-Compliant Process For Generating Highly Efficient Genome Edited HSPC

Gary K Lee, PhD, Lynn Truong, Travis Wood, Jill Henley, Lee Ya-Li, Ken Kim, Yuanyue Zhou, Jianbin Wang, Ph.D., Andreas Reik, PhD, Fyodor D Urnov, PhD, Paula Cannon, Ph.D., Michael C. Holmes, PhD, Dale Ando, M.D., Martin A Giedlin, Ph.D.

Blood (2013) 122 (21): 4213.

https://doi.org/10.1182/blood.V122.21.4213.4213

Published: 2013

Journal Articles

Using Forced Chromatin Looping To Overcome Developmental Silencing Of Embryonic and Fetal β-Type Globin Genes In Adult Erythroid Cells

Jeremy W Rupon, MD, PHD, Wulan Deng, PhD, Hongxin Wang, Philip D Gregory, PhD, Andreas Reik, PhD, Ann Dean, PhD, Gerd A. Blobel, MD, PhD

Blood (2013) 122 (21): 433.

https://doi.org/10.1182/blood.V122.21.433.433

Published: 2013

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-Driven Gene Editing Prevents HLA-A Expression On Hematopoietic Stem Cells -Improving The Chance Of Finding An HLA-Matched Donor

Hiroki Torikai, Tiejuan Mi, Sonny O Ang, PhD, Loren Gragert, Martin Maiers, Sourindra Maiti, PHD, Kirsten Switzer, Helen Huls, BS, Andreas Reik, Michael C. Holmes, PhD, Philip D Gregory, PhD, Richard E Champlin, MD, Elizabeth J Shpall, MD, Laurence J N Cooper, MD, PhD

Blood (2013) 122 (21): 1655.

https://doi.org/10.1182/blood.V122.21.1655.1655

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

Controlling Long-Range Genomic Interactions to Reprogram the β-Globin Locus

Wulan Deng, Graduate student, Jeremy W Rupon, MD, PhD, Hongxin Wang, Andreas Reik, Philip D. Gregory, PhD, Gerd A Blobel, MD, PhD

Blood (2012) 120 (21): 280.

https://doi.org/10.1182/blood.V120.21.280.280

Published: 2012

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

TCR Gene Editing Results in Effective Immunotherapy of Leukemia without the Development of GvHD

Blood (2011) 118 (21): 667.

https://doi.org/10.1182/blood.V118.21.667.667

Published: 2011

Journal Articles

Manipulating Higher Order Chromatin Structure of the β-Globin Locus by Targeted Tethering of a “looping” Factor

Wulan Deng, Graduate, student, Philip D Gregory, PhD, Andreas Reik, PhD, Gerd Blobel, MD, PhD

Blood (2010) 116 (21): 647.

https://doi.org/10.1182/blood.V116.21.647.647

Published: 2010

NARROW

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

Ex Vivo Gene-Edited Cell Therapy for Sickle Cell Disease: Disruption of the BCL11A Erythroid Enhancer with Zinc Finger Nucleases Increases Fetal Hemoglobin in Plerixafor Mobilized Human CD34+ Cells

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

NY-ESO-1 TCR single edited stem and central memory T cells to treat multiple myeloma without graft-versus-host disease

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

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

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

NY-ESO-1 Single Edited T Cells to Treat Multiple Myeloma without Inducing GvHD

TCR Gene Editing Achieved In a Single Round Of T Cell Activation Is Sufficient To Redirect T Cell Specificity and Prevent GvHD

Zinc Finger Nucleases Targeting The β-Globin Locus Drive Efficient Correction Of The Sickle Mutation In CD34 + Cells

Autologous Hematopoietic Stem/Progenitor Cell (HSPC) Therapy For Monogenic Blood Disorders: Scalable, cGMP-Compliant Process For Generating Highly Efficient Genome Edited HSPC

Using Forced Chromatin Looping To Overcome Developmental Silencing Of Embryonic and Fetal β-Type Globin Genes In Adult Erythroid Cells

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

ZFN-Driven Gene Editing Prevents HLA-A Expression On Hematopoietic Stem Cells -Improving The Chance Of Finding An HLA-Matched Donor

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

Controlling Long-Range Genomic Interactions to Reprogram the β-Globin Locus

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

TCR Gene Editing Results in Effective Immunotherapy of Leukemia without the Development of GvHD

Manipulating Higher Order Chromatin Structure of the β-Globin Locus by Targeted Tethering of a “looping” Factor

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American Society of Hematology

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American Society of Hematology

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