Targeting the EIF2AK1 Signaling Pathway Rescues Red Blood Cell Production in SF3B1-Mutant Myelodysplastic Syndromes With Ringed Sideroblasts.

Vera Adema Feiyang Ma Rashmi Kanagal-Shamana Natthakan Thongon Guillermo Montalban Bravo Hui Yang Scott A Peslak Feng Wang Pamela Acha Francisco Solé Pamela Lockyer Margherita Cassari Jaroslaw P Maciejewski Valeria Visconte Irene Gañán-Gómez Yuan-Bin Song Carlos E Bueso-Ramos Matteo Pellegrini Tuyet M Tan Rafael Bejar Jennifer S Carew Stephanie Halene Valeria Santini Gheath Al-Atrash Karen Clise-Dwyer Guillermo Garcia Manero Gerd A Blobel Simona Colla

Published 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.

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