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David Justus
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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

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

Evaluating Metabolic Regulation of Human Erythropoiesis By Profiling Nutrient Transporter Expression

Free
David Justus, MD, Hongxia Yan, MS, John Hale, PhD, Lionel Blanc, PhD, Floriane Bitaudeau, MS, Vincent Petit, DVM, PhD, Sandrina Kinet, PhD, Marc Sitbon, PhD, Naomi Taylor, MD PhD, Mohandas Narla, DSc
Journal: Blood
Blood (2019) 134 (Supplement_1): 342.
https://doi.org/10.1182/blood-2019-124380
Published: 2019