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John Manis
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Journal Articles

Efficient, potent, and specific near-universal base and prime editing for ELANE-mutant severe congenital neutropenia

Free
Jing Zeng, Nola Neri, Josias Soares de Brito, Archana Verma, Sébastien Levesque, Gue-Ho Hwang, Viktoria Jung, Gabriele Casirati, Felicia Andresen, David Justus, Denisa Wagner, John Manis, Danilo Pellin, Pietro Genovese, Akiko Shimamura, Myriam Armant, Scot Wolfe, Peter Newburger, Daniel Bauer
Journal: Blood
Blood (2025) 146 (Supplement 1): 624.
https://doi.org/10.1182/blood-2025-624
Published: 2025
Journal Articles

Apheresis blood processing with heparin improves stem cell collection outcomes for sickle cell gene therapy

Free
David Justus, Carly Howard, Ellen Proeung, Aimee Sarnie, Jen Fortin, Erica Esrick, Matthew Heeney, Daniel Bauer, Sarah Nikiforow, Jose Cancelas, Leslie Lehmann, David Williams, John Manis
Journal: Blood
Blood (2025) 146 (Supplement 1): 6096.
https://doi.org/10.1182/blood-2025-6096
Published: 2025
Journal Articles

Highly efficient collection and manufacture of autologous HSC gene therapy cell product for patients with sickle cell disease using a lentiviral vector containing a shmir targeting BCL11a

Free
John Manis, David Justus, Erica Esrick, Edmund Waller, Mary Horowitz, Leslie Lehmann, Amy Federico, Colleen Dansereau, Emily Morris, Leah Cheng, Mehrdad Abedi, Michael Pulsipher, Neena Kapoor, Gary Schiller, Angela Treml, Sonali Chaudhury, Victoria Coleman-Cowger, Kit Shaw, Jerome Ritz, Heather Daley, Donald Kohn, Akshat Jain, Jeanne Hendrickson, Carly Howard, Aimee Sarnie, Ellen Proeung, Helene Vincon, Myriam Armant, Joseph Antin, Mark Walters, Mary Eapen, David Williams
Journal: Blood
Blood (2025) 146 (Supplement 1): 6094.
https://doi.org/10.1182/blood-2025-6094
Published: 2025
Journal Articles

Efficient prime editing of human hematopoietic stem cells corrects the recurrent SBDS C.258+2T>C mutation and recovers engraftment potential in shwachman-diamond syndrome

Free
Gue-Ho Hwang, Michael Peters, Felicia Andresen, Jing Zeng, Haarika Kathi, Sébastien Levesque, Chad Harris, Christy Kolsteeg, Nola Neri, Archana Verma, Eva Wauters, Chi Yuan Zhang, Myriam Armant, Christian Brendel, Scot Wolfe, John Manis, Akiko Shimamura, Daniel Bauer
Journal: Blood
Blood (2025) 146 (Supplement 1): 1063.
https://doi.org/10.1182/blood-2025-1063
Published: 2025
Journal Articles

Efficacy of motixafortide plus G-CSF versus plerixafor plus G-CSF for stem cell mobilization and collection in multiple myeloma: A single-center comparative analysis and interim Results from a prospective study in poor mobilizers

Free
Sara Close, Jun Liu, Lara Abousamra, Suhayla Sarhan, Kristine Geary, Sanjana Arji, Claire Shao, Eileen Green, Yuxin Liu, Paul Richardson, John Manis, Corey Cutler, Sarah Nikiforow, Clifton Mo
Journal: Blood
Blood (2025) 146 (Supplement 1): 4185.
https://doi.org/10.1182/blood-2025-4185
Published: 2025
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

Long-term follow-up of the first in human post-transcriptional genetic silencing of BCL11A in sickle cell disease in a phase 1 pilot and feasibility trial

Free
Erica Esrick, Leslie Lehmann, Amy Federico, Heather Daley, Colleen Dansereau, Satiro DeOliveira, John Everett, Pei-Chi Kao, Boya Liu, Theodore Moore, Emily Morris, Helene Negre, Kit Shaw, Gavin Roach, Jerome Ritz, Aoife Doto, Olivia Silva, Helene Vincon, Frederic Bushman, Donald Kohn, Akshat Jain, Wendy London, David Justus, Myriam Armant, John Manis, David Williams
Journal: Blood
Blood (2025) 146 (Supplement 1): 2533.
https://doi.org/10.1182/blood-2025-2533
Published: 2025
Journal Articles

Improved hematopoietic stem cell mobilization for gene therapy using single agent motixafortide in sickle cell disease

Free
Stephan Grupp, Michael Grimley, Timothy Driscoll, Kris Mahadeo, Alexis Leonard, Yongping Wang, Helen Phillips, David Justus, Leslie Lehmann, Erica Esrick, Julie Kanter, Sarah Nikiforow, Matthew Hsieh, John Tisdale, Akshay Sharma, Zachary Crees, John Manis
Journal: Blood
Blood (2025) 146 (Supplement 1): 2542.
https://doi.org/10.1182/blood-2025-2542
Published: 2025
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

UM171 Enhances Fitness and Engraftment of Gene Modified Hematopoietic Stem Cells from Sickle Cells Disease Patients

Free
Boya Liu, Denise Klatt, Yu Zhou, John Manis, Guy Sauvageau, Christian Brendel, David A. Williams
Journal: Blood
Blood (2023) 142 (Supplement 1): 18.
https://doi.org/10.1182/blood-2023-182099
Published: 2023
Journal Articles

B cell–intrinsic deficiency of the Wiskott-Aldrich syndrome protein (WASp) causes severe abnormalities of the peripheral B-cell compartment in mice

Free
Mike Recher, Siobhan O. Burns, Miguel A. de la Fuente, Stefano Volpi, Carin Dahlberg, Jolan E. Walter, Kristin Moffitt, Divij Mathew, Nadine Honke, Philipp A. Lang, Laura Patrizi, Hervé Falet, Marton Keszei, Masayuki Mizui, Eva Csizmadia, Fabio Candotti, Kari Nadeau, Gerben Bouma, Ottavia M. Delmonte, Francesco Frugoni, Angela B. Ferraz Fomin, David Buchbinder, Emma Maria Lundequist, Michel J. Massaad, George C. Tsokos, John Hartwig, John Manis, Cox Terhorst, Raif S. Geha, Scott Snapper, Karl S. Lang, Richard Malley, Lisa Westerberg, Adrian J. Thrasher, Luigi D. Notarangelo
Journal: Blood
Blood (2012) 119 (12): 2819–2828.
https://doi.org/10.1182/blood-2011-09-379412
Published: 2012
Includes: Supplemental data
Journal Articles

FAK Regulates Progenitor B Cell Growth, Localization and Retention in Bone Marrow Microenvironments

Free
Shin-Young Park, Peter Wolfram, John Manis, Hilary Beggs, Cesar Nombela-Arrieta, Gregory Pivarnik, Leslie E Silberstein, MD
Journal: Blood
Blood (2011) 118 (21): 1119.
https://doi.org/10.1182/blood.V118.21.1119.1119
Published: 2011
Journal Articles

The BBAP E3 Ligase Monoubiquitylates Histone H4 at Lysine 91 and Selectively Modulates the DNA Damage Response in Chemotherapy-Resistant Lymphomas.

Free
Qingsheng Yan, PH.D., Shilpee Dutt, Ph.D., Rong Xu, Katherine Graves, Przemyslaw Juszczynski, MD, PhD, John Manis, MD, Margaret A. Shipp, MD
Journal: Blood
Blood (2009) 114 (22): 3958.
https://doi.org/10.1182/blood.V114.22.3958.3958
Published: 2009
Journal Articles

Ott1 (Rbm15) Is Required for the Large to Small Pre B-Cell Transition

Free
Michael Kharas, John Manis, MD, D. Gary Gilliland, MD, PhD, Glen D. Raffel, MD, PhD
Journal: Blood
Blood (2008) 112 (11): 706.
https://doi.org/10.1182/blood.V112.11.706.706
Published: 2008
Journal Articles

53BP1 Is Targeted to Sites of DNA Breaks within the Immunoglobulin Heavy Chain Locus and Promotes Class Switch Recombination.

Free
John Manis, MD, Nicole Walsh, BA, Phil Carpenter, PhD, Shilpee Dutt, PhD
Journal: Blood
Blood (2006) 108 (11): 2379.
https://doi.org/10.1182/blood.V108.11.2379.2379
Published: 2006