While suppression of Hh pathway by SMO inhibitors such as cyclopamine has been used as a therapeutic strategy for cancer, a significant fraction of GLI1 activation in PDA is driven by a SMO-independent mechanism [19], suggesting that direct inhibition of GLI1 protein may be a more effective route to suppress Hh pathway activation [20], [21] in PDA. The gene discussed is GLI1; the disease is Patent ductus arteriosus.