Evidences favoring the relevance of targeting PI3K/mTOR pathway in MPN derive from cellular and animal models [27, 28]; furthermore, in a phase I/II clinical trial in patients with myelofibrosis, the mTORC1 inhibitor RAD001 (Everolimus) demonstrated clinical effectiveness by reducing splenomegaly and improving constitutional symptoms [29]. The gene discussed is MTOR; the disease is myeloproliferative disorder.