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CNS Repopulation by Hematopoietic-Derived Microglia-Like Cells Corrects Progranulin deficiency
Pasqualina Colella1, Ruhi Sayana1, Maria Valentina Suarez-Nieto1
1Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, 94305.
Research Square
|October 4, 2023
Summary
Hematopoietic stem cell transplantation can deliver therapies to the CNS. A new conditioning regimen using Busulfan and PLX3397 enables efficient microglia replacement, showing therapeutic potential in a GRN deficiency model.
Area of Science:
- Neuroscience
- Immunology
- Hematology
Background:
- Hematopoietic stem cell transplantation (HSCT) offers potential for CNS protein delivery via microglia-like cells.
- Current HSCT conditioning methods result in slow and inefficient microglia engraftment.
Conclusions:
- This conditioning regimen significantly enhances microglia engraftment for potential CNS therapies.
- The approach demonstrates therapeutic efficacy in a relevant disease model, highlighting its clinical potential.

