title: Modulators of erythropoiesis: emerging therapies for hemoglobinopathies and disorders of red cell production.
abstract: Use of new compound such as inhibitors of JAK2 or transforming growth factor β-like molecules might soon revolutionize the treatment of β-thalassemia and related disorders. However, this situation requires careful optimization, noting the potential for off-target immune suppression for JAK2 inhibitors and the lack of mechanistic insights for the use of the ligand trap soluble molecules that sequester ligands of activin receptor IIA and B.
authors.affiliation: Department of Pediatrics, Hematology-Oncology, Weill Cornell Medical College, 1300 York Avenue, New York, NY 10021, USA. Electronic address: lab2002@med.cornell.edu.
authors.forename: Laura
authors.initials: L
authors.lastname: Breda
authors.affiliation: Department of Pediatrics, Hematology-Oncology, Weill Cornell Medical College, 1300 York Avenue, New York, NY 10021, USA; Department of Cell and Developmental Biology, Weill Cornell Medical College, 1300 York Avenue, New York, NY 10021, USA.
authors.forename: Stefano
authors.initials: S
authors.lastname: Rivella
mesh_terms: D006801:Humans; D030301:Activin Receptors, Type II / Q000037:antagonists & inhibitors / Q000378:metabolism; D017086:beta-Thalassemia / Q000188:drug therapy / Q000378:metabolism / Q000503:physiopathology; D004912:Erythrocytes / Q000187:drug effects / Q000378:metabolism / Q000502:physiology; D004920:Erythropoiesis / Q000187:drug effects / Q000502:physiology; D006453:Hemoglobinopathies / Q000188:drug therapy / Q000378:metabolism / Q000503:physiopathology; D053614:Janus Kinase 2 / Q000037:antagonists & inhibitors / Q000378:metabolism; D058990:Molecular Targeted Therapy / Q000379:methods / Q000639:trends; D011993:Recombinant Fusion Proteins / Q000494:pharmacology / Q000627:therapeutic use; D007141:Immunoglobulin Fc Fragments
chemical_list: C542017:sotatercept; D030301:Activin Receptors, Type II; D053614:Janus Kinase 2; D011993:Recombinant Fusion Proteins; C000621232:luspatercept; D007141:Immunoglobulin Fc Fragments
keywords: ACE-011; ACE-536; Hemoglobinopathies; JAK2; LY-2157299; Steady state; Stress and ineffective erythropoiesis; Transforming growth factor β