Naryshkin et al.127 found that an SMA patient-derived iPSC model could be used to validate human- and disease-specific drug responsiveness after initial screening using a HEK293 cell line127. These compounds were then validated in patient-specific fibroblasts, and in motor neurons differentiated from patient-derived iPSCs that serve as a patient-specific and disease-relevant cellular model127. Finally, the hit compound was evaluated in a mouse model for in vivo activity127. This drug discovery approach includes a patient-derived iPSC model as one of the validation steps by taking advantage of the patient-specific and disease-relevant properties of motor neurons derived from patient iPSCs.
