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Updated: Aug 6, 2026

Transplantation of Human Induced Pluripotent Stem Cell-Derived Microglia in Immunocompetent Mice Brain via Non-Invasive Transnasal Route
Published on: May 31, 2022
Induced pluripotent stem cell treatment for microglia replacement in progressive MS
Agastya Reddy Malladi1, Naghmeh Abbasi Kasbi1, Jeffrey A Cohen2
1Department of Neurology, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Induced pluripotent stem cells (iPSCs) show promise for replacing harmful microglia in multiple sclerosis (MS). While direct studies are lacking, preclinical data suggest iPSC-derived cells could be a viable therapy for MS.
Area of Science:
- Neuroscience
- Regenerative Medicine
- Immunology
Background:
- Microglia are key drivers of pathology in multiple sclerosis (MS).
- Targeting microglia with colony-stimulating factor 1 receptor (CSF-1R) inhibitors is a potential therapeutic strategy.
- Induced pluripotent stem cells (iPSCs) offer a source for cell replacement therapies.
Purpose of the Study:
- To evaluate the feasibility of using iPSC-derived microglia to replace pathogenic microglia in MS.
- To review existing literature on microglial depletion, iPSC differentiation, and cell transplantation in the CNS.
Main Methods:
- Systematic literature search.
- Review of studies on microglial depletion and CSF-1R inhibitors.
- Analysis of iPSC differentiation protocols for microglia generation.
- Examination of preclinical transplantation studies in MS models.
Main Results:
- No direct studies currently exist on iPSC-derived microglia transplantation in MS.
- Preclinical studies with other iPSC-derived neural cells show successful engraftment and functional recovery in MS models.
- Evidence supports the biological plausibility of iPSC-based CNS cell replacement.
Conclusions:
- Replacing pathogenic microglia with iPSC-derived microglia is a potential therapeutic strategy for progressive MS.
- Further preclinical research is essential to establish safety, optimize transplantation, and assess long-term efficacy.
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