In vivo MPI/BLI tracking of genome-edited-patient derived neural precursor cells in a transgenic ALS mouse model

Amyotrophic lateral sclerosis (ALS) caused by superoxide dismutase 1 (SOD1) mutations may benefit from a gene-edited induced pluripotent stem cell (iPSC)-based therapy, provided cell fate after administration can be monitored precisely, non-invasively and longitudinally for effective optimization. This study evaluated tracking of neural precursor cells (NPCs) derived from gene-edited iPSCs using magnetic particle imaging (MPI) and bioluminescence imaging (BLI). Luciferase-expressing Nanoluc-NPCs were magnetically labeled and stereotaxically transplanted into the motor cortex of SOD1G93A transgenic mouse models. BLI confirmed luciferase activity and cell viability, while MPI enabled quantitative tracking of labeled cells in vivo. Regression analysis estimated an iron content of ~10 pg per cell, with MPI-derived cell numbers aligning closely with injected values. BLI signals persisted for two weeks, and behavioral testing revealed delayed disease progression for treated vs. non-treated controls. These results demonstrate successful non-invasive visualization and quantification of transplanted NPCs, supporting the use of MPI/BLI for imaging-guided cell therapies in ALS.