Targeted therapies that suppress the STAT3 pathway (e.g., JAK inhibitors), enhance protein translation (e.g., NGF and EGF treatments), modulate the activity of the mTOR pathway (e.g., NV-5138, everolimus), and restore the SAM/SAH methylation balance (e.g., betaine treatment) hold significant promise for alleviating multiple symptoms of MDS. The gene discussed is MTOR; the disease is myelodysplastic syndrome.