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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 ?-like molecules might soon revolutionize the treatment of ?-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.

SUBMITTER: Breda L

PROVIDER: S-EPMC3970239 | biostudies-literature | 2014 Apr

REPOSITORIES: biostudies-literature

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Modulators of erythropoiesis: emerging therapies for hemoglobinopathies and disorders of red cell production.

Breda Laura L Rivella Stefano S

Hematology/oncology clinics of North America 20140118 2

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. ...[more]

PMID: 24589272

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