Injections with 2-hydroxypropyl-β-cyclodextrin are currently in phase 2/3 clinical trials for NPC1 disease (NCT02534844) and improve liver function, delay neurodegeneration, and increase life span of Npc1−/− mice (60, 61, 62) but do not decrease splenomegaly in WTD-fed Ldlr−/− mice transplanted with BM from mice with Npc1 loss-of-function (63). The gene discussed is NPC1; the disease is Splenomegaly.