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Journal Articles

Gene Therapy: Where We Started and Where the Field Is Going

Philip D. Gregory, DPhil

Blood (2017) 130 (Suppl_1): SCI-19.

https://doi.org/10.1182/blood.V130.Suppl_1.SCI-19.SCI-19

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

In Vivo Genome Editing in Neonatal Mouse Liver Preferentially Utilizes Homology Directed Repair

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

Blood (2015) 126 (23): 4422.

https://doi.org/10.1182/blood.V126.23.4422.4422

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

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

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

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

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 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

Robust ZFN-mediated genome editing in adult hemophilic mice

Xavier M. Anguela, Rajiv Sharma, Yannick Doyon, Jeffrey C. Miller, Hojun Li, Virginia Haurigot, Michelle E. Rohde, Sunnie Y. Wong, Robert J. Davidson, Shangzhen Zhou, Philip D. Gregory, Michael C. Holmes, Katherine A. High

Blood (2013) 122 (19): 3283–3287.

https://doi.org/10.1182/blood-2013-04-497354

Published: 2013

Includes: Supplemental data

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

In Vivo Genome Editing of Liver Albumin for Therapeutic Gene Expression: Rescue of Hemophilic Mice Via Integration of Factor 9

Xavier M Anguela, PhD, Rajiv Sharma, Yannick Doyon, PhD, Sunnie Y Wong, David E Paschon, Hojun Li, MD PhD, Virginia Haurigot, PhD, Robert J Davidson, Shangzhen Zhou, MD, Philip D. Gregory, PhD, Michael C. Holmes, PhD, Edward Rebar, PhD, Katherine A. High, MD

Blood (2012) 120 (21): 751.

https://doi.org/10.1182/blood.V120.21.751.751

Published: 2012

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

NARROW

Gene Therapy: Where We Started and Where the Field Is Going

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

In Vivo Genome Editing in Neonatal Mouse Liver Preferentially Utilizes Homology Directed Repair

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

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

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

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

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

Robust ZFN-mediated genome editing in adult hemophilic mice

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

In Vivo Genome Editing of Liver Albumin for Therapeutic Gene Expression: Rescue of Hemophilic Mice Via Integration of Factor 9

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

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American Society of Hematology

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American Society of Hematology

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