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Updated: Oct 27, 2025

Transplantation of Human Induced Pluripotent Stem Cell-Derived Microglia in Immunocompetent Mice Brain via Non-Invasive Transnasal Route
Published on: May 31, 2022
Microglia replacement by bone marrow transplantation (Mr BMT) in the central nervous system of adult mice
1Shenzhen Institute of Advanced Technology, Chinese Academy of Sciences, Shenzhen 518055, China.
Abstract:
Microglia are important immune cells in the central nervous system (CNS). Mutations in microglia may cause CNS disorders. Replacement of dysfunctional microglia with allogeneic wild-type microglia can correct the gene deficiency, thus treating the neurogenic diseases. However, traditional approaches cannot efficiently replace microglia at the adulthood. Here, we introduce a potentially clinical-feasible strategy named microglia replacement by bone marrow transplantation that achieves efficient microglia replacement at the whole CNS scale, including the brain, spinal cord, and retina in adult mice. For complete details on the use and execution of this protocol, please refer to Xu et al. (2020). The original abbreviation of this microglia replacement strategy is mrBMT. We hereby change the name to Mr BMT.
Insights
Bone marrow transplantation can replace dysfunctional microglia in the adult central nervous system (CNS). This novel approach, termed Mr BMT, offers a potential treatment for CNS disorders caused by microglial mutations.
Area of Science:
- Neuroscience
- Immunology
- Stem Cell Biology
Background:
- Microglia are crucial immune cells within the central nervous system (CNS).
- Genetic mutations affecting microglia can lead to various CNS disorders.
- Efficient microglia replacement in adult organisms is a significant challenge for therapeutic strategies.
Purpose of the Study:
- To introduce and validate a clinically feasible method for widespread microglia replacement in adult mice.
- To establish an effective strategy for correcting gene deficiencies in microglia-associated CNS diseases.
Main Methods:
- Development of a bone marrow transplantation protocol for microglia replacement (Mr BMT).
- Application of the Mr BMT strategy to achieve whole CNS microglia repopulation in adult mice.
- Assessment of microglia replacement efficacy across the brain, spinal cord, and retina.
Main Results:
- Demonstrated efficient microglia replacement throughout the entire CNS in adult mice.
- The Mr BMT strategy proved effective in achieving large-scale microglia engraftment.
- The protocol targets the brain, spinal cord, and retina, indicating broad applicability.
Conclusions:
- Microglia replacement by bone marrow transplantation (Mr BMT) is a viable and efficient strategy for the adult CNS.
- This method holds promise for treating neurogenic diseases stemming from microglial dysfunction or mutations.
- The Mr BMT approach offers a potential clinical pathway for CNS disorder therapies.

