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

Research
Written By Christine Briscoe

Authors: 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

Published: November 3, 2025

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Christine Briscoe
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The feasibility of collecting social determinants of health in the national multi-center GRASP sickle cell gene therapy trial
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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