Study on the Safety of Human Oligodendrocyte Precursor Cells Transplantation in Young Animals and Its Efficacy on Myelination

Oligodendrocyte precursor cells (OPCs), which can differentiate into myelinating oligodendrocytes during embryonic development, are an important potential source for myelin repair or regeneration. To date, OPCs from human sources (hOPCs) remain limited. In this study, we aimed to evaluate the safety and remyelination capacity of hOPCs developed in our laboratory by transplanting them into the lateral ventricles of Sprague–Dawley rats of different ages. The toxicity, biodistribution, and tumor formation abilities of the injected hOPCs were examined by evaluating rats’ vital signs, developmental indicators, neural reflexes, along with hematological, immunological, and pathological assessments. In addition, the hOPCs were transplanted into the corpus callosum of shiverer mice to verify cell myelination efficacy. Overall, our results showed that transplanted hOPCs into young mice showed no toxicity against their organ function or immune system, engrafted only in the brain, and caused no tissue proliferation or tumor formation. In terms of efficacy, the transplanted hOPCs formed myelin in the corpus callosum, alleviated the trembling phenotype of shiverer mice, and promoted normal development. The transplantation of hOPCs is safe and can effectively form myelin in the brain, thereby providing a theoretical basis for the future clinical transplantation of hOPCs.