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Michael C Holmes
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Journal Articles

In Vivo correction of the sickle cell disease mutation in hematopoietic stem cells using RNA gene writers

Free
Yaser Heshmati, Giulia Schiroli, Lorenzo Tozzi, Michael Monte, Nouhaila Beytour, Apiwat Wangweerawong, James Rottman, William E. Salomon, Rahul Palchaudhuri, Z. Jane Wang, Anne Bothmer, Kim Vanuytsel, John Manis, Lev Gorfinkel, Francesca Alvarez Calderon, Ulrike Gerdemann, Leslie Kean, Hari Pujar, Cecilia Cotta-Ramusino, Raja Prince-Eladnani, Michael C Holmes
Journal: Blood
Blood (2025) 146 (Supplement 1): 2536.
https://doi.org/10.1182/blood-2025-2536
Published: 2025
Journal Articles

In Vivo RNA delivery by targeted lipid nanoparticles enable gene editing in hematopoietic stem cells and T cells

Free
Rahul Palchaudhuri, Michael Monte, Mohit Gupta, Giulia Schiroli, Lorenzo Tozzi, Lei Liu, Han Gu, Rebecca Goldman, Apiwat Wangweerawong, Aaron Zannini, Yaser Heshmati, Christina Ferren, Alyssa Sanfilippo, Tiffany Morales, Jessica Desmond, Kartika Venugopal, Jason Rodriguez, Albert De Iaco, Cecilia Cotta-Ramusino, William E. Salomon, James Rottman, William Querbes, Hari Pujar, Michael C Holmes, Z. Jane Wang
Journal: Blood
Blood (2025) 146 (Supplement 1): 4318.
https://doi.org/10.1182/blood-2025-4318
Published: 2025
Journal Articles

Targeted lnp delivery of an RNA gene writer In Vivo enables generation of functional CAR-T cells

Free
Cecilia Cotta-Ramusino, Albert De Iaco, James Rottman, Jason Rodriguez, Donghui Li, Kartika Venugopal, Jessica Desmond, Christina Ferren, Mohit Gupta, Rebecca Levy, Rahul Palchaudhuri, Z. Jane Wang, William Querbes, Hari Pujar, Michael C Holmes
Journal: Blood
Blood (2025) 146 (Supplement 1): 6097.
https://doi.org/10.1182/blood-2025-6097
Published: 2025
Journal Articles

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

Free
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
Journal: Blood
Blood (2024) 144 (Supplement 1): 2197.
https://doi.org/10.1182/blood-2024-208968
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

Free
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
Journal: Blood
Blood (2024) 144 (Supplement 1): 4799.
https://doi.org/10.1182/blood-2024-208176
Published: 2024
Journal Articles

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

Free
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
Journal: Blood
Blood (2024) 144 (Supplement 1): 515.
https://doi.org/10.1182/blood-2024-208626
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

Free
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
Journal: Blood
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

Free
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
Journal: Blood
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)

Free
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
Journal: Blood
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

Free
Sara Mastaglio, Pietro Genovese, Zulma Magnani, Eliana Ruggiero, Elisa Landoni, Barbara Camisa, Giulia Schiroli, Elena Provasi, Angelo Lombardo, Andreas Reik, Nicoletta Cieri, Martina Rocchi, Giacomo Oliveira, Giulia Escobar, Monica Casucci, Bernhard Gentner, Antonello Spinelli, Anna Mondino, Attilio Bondanza, Luca Vago, Maurilio Ponzoni, Fabio Ciceri, Michael C. Holmes, Luigi Naldini, Chiara Bonini
Journal: Blood
Blood (2017) 130 (5): 606–618.
https://doi.org/10.1182/blood-2016-08-732636
Published: 2017
Includes: Supplemental data
Journal Articles

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

Free
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
Journal: Blood
Blood (2016) 128 (22): 3636.
https://doi.org/10.1182/blood.V128.22.3636.3636
Published: 2016
Journal Articles

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

Free
Brigit E. Riley, PhD, Jeff Boonsripisal, Alicia Goodwin, Dominique Cartier, Eudean Garces, Kate Nesbitt, Courtney Dwyer, Stephen Ballaron, Kassandra Hebert, Hung Tran, Andrea Kang, Lei Zhang, PhD, Kathleen E. Meyer, PhD, Judith Greengard, PhD, Christine Hough, PhD, Richard Surosky, PhD, Dale Genji Ando, MD, David Lillicrap, MD FRCPC, Geoff Nichol, MB, ChB, Michael C. Holmes, PhD
Journal: Blood
Blood (2016) 128 (22): 1173.
https://doi.org/10.1182/blood.V128.22.1173.1173
Published: 2016
Journal Articles

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

Free
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
Journal: Blood
Blood (2016) 127 (20): 2416–2426.
https://doi.org/10.1182/blood-2015-09-672337
Published: 2016
Includes: Supplemental data
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

Free
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
Journal: Blood
Blood (2015) 126 (23): 200.
https://doi.org/10.1182/blood.V126.23.200.200
Published: 2015
Journal Articles

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

Free
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
Journal: Blood
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

Free
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
Journal: Blood
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

Free
Megan D. Hoban, Gregory J. Cost, Matthew C. Mendel, Zulema Romero, Michael L. Kaufman, Alok V. Joglekar, Michelle Ho, Dianne Lumaquin, David Gray, Georgia R. Lill, Aaron R. Cooper, Fabrizia Urbinati, Shantha Senadheera, Allen Zhu, Pei-Qi Liu, David E. Paschon, Lei Zhang, Edward J. Rebar, Andrew Wilber, Xiaoyan Wang, Philip D. Gregory, Michael C. Holmes, Andreas Reik, Roger P. Hollis, Donald B. Kohn
Journal: Blood
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

Free
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
Journal: Blood
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

Free
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
Journal: Blood
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

Free
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
Journal: Blood
Blood (2014) 124 (21): 308.
https://doi.org/10.1182/blood.V124.21.308.308
Published: 2014