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allogeneic peripheral blood stem cell
- Development stage
- Phase 1
- Lead developer
- National Institute of Allergy and Infectious Diseases
- Modality
- Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
Allogeneic peripheral blood stem cells (PBSC) are hematopoietic stem cells collected from the blood of a donor and infused into a recipient to restore bone marrow function. In the context of the National Institute of Allergy and Infectious Diseases (NIAID) protocol for Chronic Granulomatous Disease (CGD), this therapy utilizes haploidentical (partially matched) related donors. The procedure involves a novel conditioning regimen consisting of Alemtuzumab, Busulfan, and Total Body Irradiation (TBI), followed by post-transplant Cyclophosphamide to mitigate graft-versus-host disease (GVHD). The primary goal is to achieve stable donor engraftment, thereby providing the patient with functional neutrophils capable of producing reactive oxygen species, which are deficient in CGD.
- Other names
- allogeneic PBSChaploidentical peripheral blood stem cell transplantallogeneic hematopoietic stem cell transplant
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