AI Article Synopsis
- Gene therapy for sickle cell disease is undergoing active trials, but collecting hematopoietic progenitor cells poses challenges due to potential complications from traditional mobilization methods.
- Plerixafor, a drug that inhibits the CXCR4 receptor, may offer a safer alternative by effectively mobilizing these progenitor cells without causing harmful side effects like vaso-occlusion in sickle cell patients.
- In preclinical studies on sickle cell mice, plerixafor successfully mobilized hematopoietic progenitor cells without triggering adverse cell activation or causing brain vaso-occlusion, supporting its potential use in clinical trials.
Article Abstract
Gene therapy for sickle cell disease is currently in active trials. Collecting hematopoietic progenitor cells safely and effectively is challenging, however, because granulocyte colony stimulating factor, the drug used most commonly for mobilization, can cause life-threatening vaso-occlusion in patients with sickle cell disease, and bone marrow harvest requires general anesthesia and multiple hip bone punctures. Plerixafor is an inhibitor of the CXCR4 chemokine receptor on hematopoietic progenitor cells, blocking its binding to SDF-1 (CXCL12) on bone marrow stroma. In support of a clinical trial in patients with sickle cell disease of plerixafor mobilization (NCT02193191), we administered plerixafor to sickle cell mice and found that it mobilizes hematopoietic progenitor cells without evidence of concomitant cell activation or brain vaso-occlusion.
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Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4806642 | PMC |
| http://dx.doi.org/10.1016/j.bcmd.2015.12.008 | DOI Listing |
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