Modeling Psychomotor Retardation using iPSCs from MCT8-Deficient Patients Indicates a Prominent Role for the Blood-Brain Barrier
Gad D. Vatine, Abraham Al-Ahmad, Bianca K. Barriga, Soshana Svendsen, Ariel Salim, Leslie Garcia, Veronica J. Garcia, Ritchie Ho, Nur Yucer, Tongcheng Qian, Ryan G. Lim, Jie Wu, Leslie M. Thompson, Weston R. Spivia, Zhaohui Chen, Jennifer Van Eyk, Sean P. Palecek, Samuel Refetoff, Eric V. Shusta, Clive N. Svendsen
Vatine et al. show that human iPSC-based modeling can pinpoint the origin of a neuronal disorder in the brain as a defect in transport of thyroid hormone across the blood-brain barrier, rather than in the neurons themselves.