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December 8, 2025Blood

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

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Authors

JMJohn P. ManisDJDavid G. JustusEEErica B. Esrick

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Overview

Phase I and II trials demonstrated successful mobilization and gene therapy outcomes in sickle cell disease, highlighting the importance of preparation and collection efficiency.

Key Points

  • Drug product success achieved with autologous hematopoietic stem cells using a lentiviral vector.
  • 74% of sickle cell disease patients reached target CD34+ collection with a mean recovery of 62%.
  • Mobilization involved daily plerixafor and transfusion strategies to suppress stress erythropoiesis.
  • Collection efficiency was improved through real-time monitoring and transfusion preparation.

Cite This Study

Manis et al. (2025) studied this question.

synapsesocial.com/papers/69362f714fa91c937236e17chttps://doi.org/10.1182/blood-2025-6094
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Long-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 trial2025 · 2 citations
  2. 2Real-world experience with apheresis for gene therapy in transfusion-dependent β-thalassemia: The largest single-center report2025
  3. 3Improved hematopoietic stem cell mobilization for gene therapy using single agent motixafortide in sickle cell disease2025
  4. 4Apheresis blood processing with heparin improves stem cell collection outcomes for sickle cell gene therapy2025 · 1 citations
  5. 5Motixafortide (CXCR4 inhibition) alone and in combination with natalizumab (VLA-4 inhibition) to mobilize hematopoietic stem cells for gene therapy in sickle cell disease: A first-in-human, safety and feasibility study2025 · 1 citations