Recently, researchers have used AAV vectors to deliver Npc1-encoding cDNA into Npc1−/− mice or NPC1-encoding mRNA into fibroblasts derived from NPC patients, both of which rescued NPC symptoms in these mice or cells, suggesting that gene therapy may offer a glimpse of hope for NPC patients (Table 1) [49,104]. This evidence concerns the gene NPC1 and nasopharyngeal carcinoma.