CNS, central nervous system; DG, dentate gyrus; ESC, embryonic pluripotent stem cell; GO, Gene Ontology; HD, Huntington disease; HTT, huntingtin; iPSC, induced pluripotent stem cells; KO, knockout; MSN, medium spiny neuron; NPC, neural progenitor; NSC, neural stem cells; polyQ, polyglutamine; PSC, pluripotent stem cells; SVZ, subventricular zone; WT, wild type. The gene discussed is HTT; the disease is Huntington disease.