Following these insights, we utilized the GATA1–FLI1–TAL1 lentiviral system to recapitulate macrothrombocytopenia in an hiPSC model of Bernard-Soulier Syndrome Type C. This approach successfully recapitulated the macrothrombocytopenia phenotype in vitro, which we subsequently restored through GP9-based gene therapy [42]. This evidence concerns the gene GATA1 and Macrothrombocytopenia.