Findings from an open-label single-arm phase I/IIa clinical study (NCT01461148) evaluating the safety and immunogenicity of a vaccine generated from FSP neoantigens derived from mutant AIM2, HT001, and TAF1B genes in patients with a history of LS-associated or sporadic dMMR/MSI CRCs demonstrated that vaccination was safe, well-tolerated, and induced both cellular and humoral immune responses in all vaccinated patients [173]. The gene discussed is AIM2; the disease is Leigh syndrome.