title: Protein-based therapeutic for anemia caused by dyserythropoiesis.
abstract: Major advances have been recently made in understanding the molecular determinants of dyserythropoiesis, particularly due to recent works in β-thalassemia. The purpose of this review is devoted to underline the role of some proteins recently evidenced in the field, that may be new alternative therapeutic targets in the near future to alleviate different types of anemia. Areas covered: This review covers the contemporary aspects of some proteins involved in various types of dyserythropoiesis, including the transcriptional factor GATA-1 and its protective chaperone HSP70, but also cytokines of the transforming growth factor beta (TFG-β) family, TGF-β1 and GDF-11, and hormones as erythroferrone. It will be not exhaustive, but based on major recent published works from the literature in the past three years. Expert commentary: Sotatercept and lustatercept, two activin receptor II ligand traps that block GDF-11, are candidate drugs providing therapeutic hope in different types of ineffective erythropoiesis, including myelodysplastic syndromes (MDS) and β-thalassemia. Furthermore, a new concept emerges to consider erythroid lineage in the bone marrow as an endocrine gland.
authors.affiliation: a Laboratoire INSERM UMR 1163 , CNRS ERL 8254 , Paris , France.|b Service de Médecine Interne, Faculté de Médecine Paris Descartes, Sorbonne Paris-Cité et Assistance Publique-Hôpitaux de Paris , Hôpital européen Georges Pompidou , Paris , France.|c Imagine Institute, Assistance Publique-Hôpitaux de Paris, Hôpital Necker , Université Paris Descartes, Sorbonne Paris Cité , Paris , France.|d Laboratory of Excellence GR-Ex , Paris , France.
authors.forename: Jean-Benoît
authors.initials: JB
authors.lastname: Arlet
authors.affiliation: a Laboratoire INSERM UMR 1163 , CNRS ERL 8254 , Paris , France.|c Imagine Institute, Assistance Publique-Hôpitaux de Paris, Hôpital Necker , Université Paris Descartes, Sorbonne Paris Cité , Paris , France.|d Laboratory of Excellence GR-Ex , Paris , France.
authors.forename: Flavia
authors.initials: F
authors.lastname: Guillem
authors.affiliation: a Laboratoire INSERM UMR 1163 , CNRS ERL 8254 , Paris , France.|c Imagine Institute, Assistance Publique-Hôpitaux de Paris, Hôpital Necker , Université Paris Descartes, Sorbonne Paris Cité , Paris , France.|d Laboratory of Excellence GR-Ex , Paris , France.|e Service d'Hématologie, Faculté de Médecine Paris Descartes , Sorbonne Paris-Cité et Assistance Publique-Hôpitaux de Paris Hôpital Necker , Paris , France.
authors.forename: Mathilde
authors.initials: M
authors.lastname: Lamarque
authors.affiliation: a Laboratoire INSERM UMR 1163 , CNRS ERL 8254 , Paris , France.|c Imagine Institute, Assistance Publique-Hôpitaux de Paris, Hôpital Necker , Université Paris Descartes, Sorbonne Paris Cité , Paris , France.|d Laboratory of Excellence GR-Ex , Paris , France.
authors.forename: Michael
authors.initials: M
authors.lastname: Dussiot
authors.affiliation: a Laboratoire INSERM UMR 1163 , CNRS ERL 8254 , Paris , France.|c Imagine Institute, Assistance Publique-Hôpitaux de Paris, Hôpital Necker , Université Paris Descartes, Sorbonne Paris Cité , Paris , France.|d Laboratory of Excellence GR-Ex , Paris , France.
authors.forename: Thiago
authors.initials: T
authors.lastname: Maciel
authors.affiliation: a Laboratoire INSERM UMR 1163 , CNRS ERL 8254 , Paris , France.|c Imagine Institute, Assistance Publique-Hôpitaux de Paris, Hôpital Necker , Université Paris Descartes, Sorbonne Paris Cité , Paris , France.|d Laboratory of Excellence GR-Ex , Paris , France.
authors.forename: Ivan
authors.initials: I
authors.lastname: Moura
authors.affiliation: a Laboratoire INSERM UMR 1163 , CNRS ERL 8254 , Paris , France.|c Imagine Institute, Assistance Publique-Hôpitaux de Paris, Hôpital Necker , Université Paris Descartes, Sorbonne Paris Cité , Paris , France.|d Laboratory of Excellence GR-Ex , Paris , France.|e Service d'Hématologie, Faculté de Médecine Paris Descartes , Sorbonne Paris-Cité et Assistance Publique-Hôpitaux de Paris Hôpital Necker , Paris , France.
authors.forename: Olivier
authors.initials: O
authors.lastname: Hermine
authors.affiliation: a Laboratoire INSERM UMR 1163 , CNRS ERL 8254 , Paris , France.|c Imagine Institute, Assistance Publique-Hôpitaux de Paris, Hôpital Necker , Université Paris Descartes, Sorbonne Paris Cité , Paris , France.|d Laboratory of Excellence GR-Ex , Paris , France.
authors.forename: Geneviève
authors.initials: G
authors.lastname: Courtois
keywords: GDF-11; HSP70; Thalassemia; dyserythropoiesis; erythroferrone; hepcidin; ineffective erythropoiesis