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
PubMed
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.