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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

Preservation of Gene Edited Hematopoietic Stem Cells By Transient Overexpression of BCL-2 mRNA

Carmen Flores Bjurström, PhD, Michelle Mojadidi, MSc, Anastasia Lomova, BS, Stephen Lai, Sorel Fitz-Gibbon, PhD, Matteo Pellegrini, PhD, Aaron R Cooper, PhD, Georgia R Lill, MSc, Michael Kaufman, MSc, Gregory J Cost, PhD, Michael C. Holmes, PhD, Roger P Hollis, PhD, Donald B Kohn

Blood (2016) 128 (22): 3636.

https://doi.org/10.1182/blood.V128.22.3636.3636

Published: 2016

Journal Articles

Engineering T-Cells Beyond Chimeric Antigen Receptor

Chiara Bonini, MD

Blood (2016) 128 (22): SCI-13.

https://doi.org/10.1182/blood.V128.22.SCI-13.SCI-13

Published: 2016

Journal Articles

The clinical applications of genome editing in HIV

Cathy X. Wang, Paula M. Cannon

Blood (2016) 127 (21): 2546–2552.

https://doi.org/10.1182/blood-2016-01-678144

Published: 2016

Journal Articles

Customizing the genome as therapy for the β-hemoglobinopathies

Matthew C. Canver, Stuart H. Orkin

Blood (2016) 127 (21): 2536–2545.

https://doi.org/10.1182/blood-2016-01-678128

Published: 2016

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

RNA Polymerase I Inhibition with CX-5461 As a Novel Strategy to Target MYC in Multiple Myeloma

Hans Lee, MD, Hua Wang, PhD, Heather Lin, M.S., Veera Baladandayuthapani, PhD, Jin He, Richard Julian Jones, PhD, Isere Kuiatse, PhD, Dongmin Gu, MD, PhD, Zhiqiang Wang, PhD, Sean O'Brien, PhD, Jonathan J Keats, PhD, Jing Yang, PhD, Richard Eric Davis, MD, Robert Z. Orlowski, PhD, MD

Blood (2015) 126 (23): 3010.

https://doi.org/10.1182/blood.V126.23.3010.3010

Published: 2015

Journal Articles

Single-Cell-State Culture of Human Pluripotent Stem Cells Increases Transfection Efficiency

Takenobu Nii, PhD, Hiroshi Kohara, PhD, Tomotoshi Marumoto, MD PhD, Tetsushi Sakuma, PhD, Takashi Yamamoto, PhD, Kenzaburo Tani, MD PhD

Blood (2015) 126 (23): 2037.

https://doi.org/10.1182/blood.V126.23.2037.2037

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

Megatal, Crispr/Cas9, and Talen T-Cell Receptor Gene Editing

Mark J. Osborn, PhD, Beau R Webber, PhD, Friederike Knipping, Cara-lin Lonetree, Anthony DeFeo, Amber McElroy, Colby Starker, PhD, Richard Gabriel, Sarah Merkel, Keli L. Hippen, PhD, Daniel Voytas, PhD, Christof von Kalle, MD, Manfred Schmidt, PhD, Jeffrey S. Miller, MD, Jordan Jarjour, PhD, Alexander Astrakhan, PhD, Andrew M Scharenberg, MD, Jakub Tolar, MD, PhD, Bruce R. Blazar, MD

Blood (2015) 126 (23): 2045.

https://doi.org/10.1182/blood.V126.23.2045.2045

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

HPRT As a Selectable Safe Harbor for Transgenesis

Anthony Conway, PhD, Josée Laganière, PhD, David E Paschon, PhD, Katrin Hacke, PhD, Noriyuki Kasahara, MD PhD, Philip D Gregory, PhD, Michael C. Holmes, PhD, Gregory J Cost, PhD

Blood (2014) 124 (21): 4796.

https://doi.org/10.1182/blood.V124.21.4796.4796

Published: 2014

Journal Articles

Gene Editing of CCR5 in Hematopoietic Stem Cells in a Nonhuman Primate Model of HIV/AIDS

Christopher W. Peterson, PhD, Jianbin Wang, PhD, Patricia Polacino, PhD, Michael C. Holmes, PhD, Shiu-Lok Hu, PhD, Philip D Gregory, PhD, Hans-Peter Kiem, MD

Blood (2014) 124 (21): 4802.

https://doi.org/10.1182/blood.V124.21.4802.4802

Published: 2014

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

Taking it to the Clinic: Genome Editing for Blood Disorders

Fyodor D. Urnov, PhD

Blood (2014) 124 (21): SCI-12.

https://doi.org/10.1182/blood.V124.21.SCI-12.SCI-12

Published: 2014

Journal Articles

Genome Editing Toolbox

Andrew M. Scharenberg, MD

Blood (2014) 124 (21): SCI-11.

https://doi.org/10.1182/blood.V124.21.SCI-11.SCI-11

Published: 2014

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)

Preservation of Gene Edited Hematopoietic Stem Cells By Transient Overexpression of BCL-2 mRNA

Engineering T-Cells Beyond Chimeric Antigen Receptor

The clinical applications of genome editing in HIV

Customizing the genome as therapy for the β-hemoglobinopathies

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

RNA Polymerase I Inhibition with CX-5461 As a Novel Strategy to Target MYC in Multiple Myeloma

Single-Cell-State Culture of Human Pluripotent Stem Cells Increases Transfection Efficiency

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

Megatal, Crispr/Cas9, and Talen T-Cell Receptor Gene Editing

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

HPRT As a Selectable Safe Harbor for Transgenesis

Gene Editing of CCR5 in Hematopoietic Stem Cells in a Nonhuman Primate Model of HIV/AIDS

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

Taking it to the Clinic: Genome Editing for Blood Disorders

Genome Editing Toolbox

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American Society of Hematology

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American Society of Hematology

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