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Promoting donor microglial replacement through augmented conditioning or radiation sensitivity.

Troy C Lund1, Willa Durose1, Emma Schindhelm1

  • 1Department of Pediatrics, Division of Pediatric Blood and Marrow Transplantation & Cellular Therapy, Minneapolis, MN 55455, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|April 13, 2026
PubMed
Summary
This summary is machine-generated.

Hematopoietic cell transplant (HCT) can engraft donor myeloid cells in the brain. Busulfan preconditioning significantly improves brain engraftment after HCT while reducing mortality, offering a safer alternative.

Keywords:
DNA damagebrainhematopoietic cell transplantationmicrogliaradiation

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Area of Science:

  • Neuroscience
  • Immunology
  • Hematology

Background:

  • Donor myeloid cells can engraft the brain post-hematopoietic cell transplant (HCT), partially replacing resident microglia.
  • Understanding conditions that promote or maintain brain engraftment is crucial for therapeutic applications.
  • Previous studies showed high brain engraftment with radiation conditioning but also high mortality.

Purpose of the Study:

  • To investigate methods for improving brain engraftment after HCT with reduced mortality.
  • To explore the role of preconditioning agents and microglia inhibition in enhancing donor cell engraftment in the brain.

Main Methods:

  • Compared busulfan preconditioning with radiation preconditioning for double HCT in mice.
  • Assessed donor myeloid cell brain engraftment in NSG and NBSGW mouse models using varying radiation doses.
  • Investigated the effect of PLX3397 (microglia inhibitor) on brain engraftment.

Main Results:

  • Busulfan preconditioning achieved 70-80% brain engraftment with only 8% mortality, unlike radiation conditioning (70% mortality).
  • Low-dose radiation (450 cGy) increased donor myeloid brain engraftment in NSG mice (15%) and NBSGW mice (>10-fold increase).
  • Pretreatment with PLX3397 further enhanced brain engraftment (median 22.5%), indicating niche clearance is beneficial.

Conclusions:

  • Preconditioning strategies, such as busulfan, are vital for successful brain engraftment after HCT.
  • Microglial niche clearance, potentially via inhibitors like PLX3397, significantly promotes donor cell engraftment in the brain.
  • Developing preconditioning-free methods for microglia replacement is essential to avoid toxicity.