Gorlin syndrome (GS) is a rare autosomal dominant disease that affects approximately 1 in 31,000 individuals worldwide. GS is primarily associated with a mutation in the PTCH1, PTCH2, or SUFU genes and may have high morbidity due to numerous phenotypic abnormalities, including palmar pits, keratocystic odontogenic tumors of the jaw bones, and most prominently, the development of multiple basal cell carcinomas (BCCs), which occur mostly on the face, head, and neck (fig 1 and 2).1,2 For most individuals, BCCs are the most difficult problem.3 The burden of disease is very high in individuals with GS due to unrelenting numbers of BCCs and other syndrome-related manifestations, as well as numerous direct and indirect costs of intervention. In addition, all currently available treatments cause pain, scarring, and/or disruptive side effects.