Liu et al demonstrated the preclinical therapeutic efficacy of mTOR inhibitor Rapamycin in the mouse model of Ptpn11 mutation-induced MPN and also in PTPN11 mutation-positive JMML patient cells.108 Likewise Parkin et al showed that AML blasts that do not express NF1 display differential sensitivity to in vitro treatment with Rapamycin.64 Here, NF1 is linked to juvenile myelomonocytic leukemia.