Neuronal and cardiac cells are primary targets of frataxin deficiency and generating models via differentiation of induced pluripotent stem cells (iPSCs) into these cell types is essential for progress towards developing therapies for FA. This review is focused on modeling FA using human iPSCs and various iPSC-differentiated cell types. The authors emphasize the importance of patient and corrected isogenic cell line pairs to minimize effects caused by biological variability between individuals.
The versatility of iPSC-derived cellular models of FA is advantageous for developing new therapeutic strategies, and rigorous testing in such models will be critical for approval of the first treatment for FA. Creating a well-characterized and diverse set of iPSC lines, including appropriate isogenic controls, will facilitate achieving this goal. Also, improvement of differentiation protocols, especially towards proprioceptive sensory neurons and organoid generation, is necessary to utilize the full potential of iPSC technology in the drug discovery process.
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