Over 80% of rare diseases are genetically inherited, with most classified as monogenic disorders caused by defects in a single gene. Advances in induced pluripotent stem cell (iPSC) technology have provided a powerful platform for recapitulating disease pathways and studying human disease pathology at a cellular level. The groundbreaking discovery of iPSCs was made possible by reprogramming somatic cells using four transcription factors, known as the “Yamanaka factors”: KLF4, MYC, OCT4, and SOX2. This reprogramming generates embryonic stem cell-like pluripotent cells, which can be differentiated into any cell type in the human body. i...More