TY - JOUR AU - Wang Fang AU - Wang Zhe AU - Wang Fen AU - Dong Kelly AU - Zhang Jing AU - Sun Yun-juan AU - Liu Chun-feng AU - Xing Mei-jie AU - Cheng Xue AU - Wei Su AU - Zheng Jia-wei AU - Zhao Xiong-fei AU - Wang Xiao-ming AU - Fu Jie AU - Song Hai-feng PY - 2020 TI - Comparative strategies for stem cell biodistribution in a preclinical study JF - Acta Pharmacologica Sinica; Vol 41, No 4 (April 2020): Acta Pharmacologica Sinica (Special Feature on Drug Research and Diagnosis of Parkinson’s Disease) Y2 - 2020 KW - N2 - Stem cell therapy represents the potential alternative effective strategy for some diseases that lack effective treatment currently. Correspondingly, it is crucial to establish high-sensitive and reliable quantification assay for tracing exogenous cell migration. In the present study, we first used both bioluminescence imaging (BLI) indirect labeling (human norepinephrine transporter-luciferase reporter system) and 89zirconium (89Zr)-hNSCs direct labeling combined with positron emission tomography/computer tomography (PET/CT) system for tracking human neural stem cells (hNSCs) migration into the brain via nasal administration in preclinical study. But the above two methods failed to give the biodistribution profile due to their low sensitivity. Considering its superior sensitivity and absolute quantitation capability, we developed and validated the droplet digital PCR (ddPCR) targeting species-specific gene in frozen and paraffin sections, slices, and whole blood with the sensitivity of 100–200 hNSCs. Accurate and high throughput quantification could be performed using ddPCR with the coefficient of variation (CVs) of lower quality control (LQC) below 30%. In combination with immunohistochemistry and ddPCR, we confirmed the migration of hNSCs into the brain via nasal administration, which supported the efficacy of hNSCs in MPTP-treated mice, an animal model of Parkinson’s disease. In conclusion, the present study is the first to report the application of ddPCR in the pharmacokinetics profile description of tracking of hNSCs in preclinical studies. UR - http://www.chinaphar.com/article/view/10129