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Journal Articles

Delivery of Gene Writers In Vivo to Hematopoietic Stem Cells and T Cells Using Targeted and Untargeted Lipid Nanoparticles (LNP)

Michael T. Monte, Lorenzo Tozzi, Rahul Palchaudhuri, Apiwat Wangweerawong, Yaser Heshmati, Giulia Schiroli, Han Gu, Yu Cao, William E. Salomon, Anne Bothmer, Jason Rodriguez, Mohit Gupta, James B. Rottman, William Querbes, Cecilia Cotta-Ramusino, Michael C Holmes, Hari Pujar, Z. Jane Wang

Blood (2024) 144 (Supplement 1): 2197.

https://doi.org/10.1182/blood-2024-208968

Published: 2024

Journal Articles

In Vivo HSC Gene Editing for Correction of the Sickle Cell Mutation Using RNA Gene Writers

Lorenzo Tozzi, Giulia Schiroli, Yaser Heshmati, Yu Cao, Michael T. Monte, Apiwat Wangweerawong, James B. Rottman, Rahul Palchaudhuri, Jean-Antoine Ribeil, John Manis, Hari Pujar, William Querbes, Anne Bothmer, Cecilia Cotta-Ramusino, Z. Jane Wang, William E. Salomon, Michael C Holmes

Blood (2024) 144 (Supplement 1): 515.

https://doi.org/10.1182/blood-2024-208626

Published: 2024

Journal Articles

Targeted Lipid Nanoparticle Delivery of an RNA Gene Writer In Vivo Enables Generation of CAR-T Cells in a Humanized Mouse Model

Jason Rodriguez, Alberto De Iaco, James B. Rottman, Donghui Liu, Kartika Venugopal, Xin Qiao, Yu Cao, Rebecca Levy, Mohit Gupta, Rahul Palchaudhuri, Bill Querbes, Cecilia Cotta-Ramusino, Hari Pujar, Madhusudan V Peshwa, Michael C Holmes

Blood (2024) 144 (Supplement 1): 4799.

https://doi.org/10.1182/blood-2024-208176

Published: 2024

Journal Articles

Deploying an RNA-Based Gene Writer System and Lipid Nanoparticle (LNP) Delivery to Generate Functional Chimeric Antigen Receptor (CAR) T Cells with in Vitro and In Vivo Anti-Tumor Activity

Michael S Magee, Alberto De Iaco, Jason Rodriguez, James B. Rottman, Donghui Liu, Giulia Schiroli, Pooja Sabhachandani, Yu Cao, Rebecca Levy, Mohit Gupta, Michael T. Monte, Jane Wang, Bill Querbes, Cecilia Cotta-Ramusino, Madhusudan V Peshwa, Michael C Holmes

Blood (2023) 142 (Supplement 1): 2073.

https://doi.org/10.1182/blood-2023-184755

Published: 2023

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

Development of an Optimized rAAV2/6 Human Factor 8 cDNA Vector Cassette for Hemophilia a Gene Therapy

Blood (2016) 128 (22): 1173.

https://doi.org/10.1182/blood.V128.22.1173.1173

Published: 2016

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

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

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

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

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

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

Simultaneous zinc-finger nuclease editing of the HIV coreceptors ccr5 and cxcr4 protects CD4 + T cells from HIV-1 infection

Chuka A. Didigu, Craig B. Wilen, Jianbin Wang, Jennifer Duong, Anthony J. Secreto, Gwenn A. Danet-Desnoyers, James L. Riley, Phillip D. Gregory, Carl H. June, Michael C. Holmes, Robert W. Doms

Blood (2014) 123 (1): 61–69.

https://doi.org/10.1182/blood-2013-08-521229

Published: 2014

Includes: Supplemental data

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

NARROW

Delivery of Gene Writers In Vivo to Hematopoietic Stem Cells and T Cells Using Targeted and Untargeted Lipid Nanoparticles (LNP)

In Vivo HSC Gene Editing for Correction of the Sickle Cell Mutation Using RNA Gene Writers

Targeted Lipid Nanoparticle Delivery of an RNA Gene Writer In Vivo Enables Generation of CAR-T Cells in a Humanized Mouse Model

Deploying an RNA-Based Gene Writer System and Lipid Nanoparticle (LNP) Delivery to Generate Functional Chimeric Antigen Receptor (CAR) T Cells with in Vitro and In Vivo Anti-Tumor Activity

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

Development of an Optimized rAAV2/6 Human Factor 8 cDNA Vector Cassette for Hemophilia a Gene Therapy

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

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

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

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

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

HPRT As a Selectable Safe Harbor for Transgenesis

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

Simultaneous zinc-finger nuclease editing of the HIV coreceptors ccr5 and cxcr4 protects CD4 + T cells from HIV-1 infection

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

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American Society of Hematology

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ASH Publications

American Society of Hematology

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