The study reports the derivation and differentiation of patient-specific induced pluripotent stem cells (iPSCs) from individuals with familial dysautonomia (FD), a rare and fatal peripheral neuropathy caused by a mutation in the *IKBKAP* gene. The researchers successfully generated two independent clones of FD-iPSCs and differentiated them into cells of all three germ layers, including peripheral neurons. Gene expression analysis revealed tissue-specific mis-splicing of *IKBKAP* in purified FD-iPSC-derived lineages. Patient-specific neural crest precursors expressed particularly low levels of normal *IKBKAP* transcript, suggesting a mechanism for disease specificity. Transcriptome analysis and cell-based assays further characterized FD pathogenesis, revealing defects in neurogenic differentiation and migration behavior. The study also validated the use of FD-iPSCs for drug screening, demonstrating that the plant hormone kinetin can reduce the mutant *IKBKAP* splice form and increase normal *IKBKAP* levels, potentially reversing aberrant splicing and improving neuronal differentiation and migration. This research highlights the potential of iPSC technology in gaining novel insights into human disease pathogenesis and treatment.The study reports the derivation and differentiation of patient-specific induced pluripotent stem cells (iPSCs) from individuals with familial dysautonomia (FD), a rare and fatal peripheral neuropathy caused by a mutation in the *IKBKAP* gene. The researchers successfully generated two independent clones of FD-iPSCs and differentiated them into cells of all three germ layers, including peripheral neurons. Gene expression analysis revealed tissue-specific mis-splicing of *IKBKAP* in purified FD-iPSC-derived lineages. Patient-specific neural crest precursors expressed particularly low levels of normal *IKBKAP* transcript, suggesting a mechanism for disease specificity. Transcriptome analysis and cell-based assays further characterized FD pathogenesis, revealing defects in neurogenic differentiation and migration behavior. The study also validated the use of FD-iPSCs for drug screening, demonstrating that the plant hormone kinetin can reduce the mutant *IKBKAP* splice form and increase normal *IKBKAP* levels, potentially reversing aberrant splicing and improving neuronal differentiation and migration. This research highlights the potential of iPSC technology in gaining novel insights into human disease pathogenesis and treatment.