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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
05:35

Transplantation of Human Induced Pluripotent Stem Cell-Derived Microglia in Immunocompetent Mice Brain via Non-Invasive Transnasal Route

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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.

Multiple Sclerosis (Houndmills, Basingstoke, England)
|July 21, 2026
PubMed
Abstract

Insights

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.
Keywords:
Multiple sclerosisinduced pluripotent stem cellsmicrogliaprogressive multiple sclerosisstem cell transplantation

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