In vitro and vivo studies showed that LDE225 (sonidegib), a highly selective and potent Smo inhibitor, was effective alone and in combination with TKIs in eradicating Hh-mediated self-renewal capacity of CD34+ and BCR-ABL1+ CML cells [18,177]. The gene discussed is CD34; the disease is chronic myelogenous leukemia, BCR-ABL1 positive.