Recent innovations in skull base approaches have permitted neurosurgeons to remove tumors that were once thought unresectable. Nevertheless, significant numbers of patients develop disabling neurologic deficits postoperatively. There remains ample opportunity for alternative treatments that are minimally invasive, and yet reduce considerably or eradicate tumor burden. Gene therapy offers such an alternative modality. Although clinical investigation has focussed primarily on malignant neoplasms, gene therapy techniques may prove effective in the treatment of benign tumors as well. Current concepts in gene therapy are discussed with specific reference to central nervous system neoplasia.