Fig. 3. Potential applications of patient-specific induced pluripotent stem cells for patients harboring ryanopathies.

Recapitulative scheme illustrating the potential use of hiPSC generated from a patient blood sample or somatic cells and carrying RyR2 mutation. Some isogenic control hiPSC could be generated by correcting the single-RyR2-point mutation using CRISPR/Cas9 technology. The hiPSC could then be differentiated into cardiomyocytes, neural and pancreatic cells. These generated cells could be used for disease modeling and drug screening approaches for a potential patient-specific cell therapy.