Primary cilium is present: correct ciliogenesis Absence of primary cilium: incorrect ciliogenesis   Will GliA be produced in the cilium? Gli target genes Cell growth Therapy Will GliA be produced in the cilium? Gli target genes Cell growth Therapy ligand overexpression (SHH, DHH, IHH) YES ON increased robotnikinin NO OFF Not increased Not required SMO activating mutations YES ON increased cyclopamine vismodegib NO OFF Not increased Not required PTCH inactivating mutations YES ON increased cyclopamine vismodegib NO OFF Not increased Not required Gli1 gene amplification NOT,  but Gli1 proteins overexpressed from Gli1 gene amplification will be the GliA forms ON increased GANT NO, but Gli1 proteins overexpressed from Gli1 gene amplification will be ON increased GANT

Table 1: Hh pathway regulation in two different conditions (with and without primary cilium): implications for specific treatments.