Targeting the EIF2AK1 Signaling Pathway Rescues Red Blood Cell Production in SF3B1-Mutant Myelodysplastic Syndromes With Ringed Sideroblasts.
Vera AdemaFeiyang MaRashmi Kanagal-ShamanaNatthakan ThongonGuillermo Montalban BravoHui YangScott A PeslakFeng WangPamela AchaFrancisco SoléPamela LockyerMargherita CassariJaroslaw P MaciejewskiValeria VisconteIrene Gañán-GómezYuan-Bin SongCarlos E Bueso-RamosMatteo PellegriniTuyet M TanRafael BejarJennifer S CarewStephanie HaleneValeria SantiniGheath Al-AtrashKaren Clise-DwyerGuillermo Garcia ManeroGerd A BlobelSimona CollaPublished in: Blood cancer discovery (2022)
MDS-RS are characterized by significant anemia. Patients with MDS-RS die from a shortage of red blood cells and the side effects of iron overload due to their constant need for transfusions. Our study has implications for the development of therapies to achieve long-lasting hematologic responses. This article is highlighted in the In This Issue feature, p. 476.