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Hematol Oncol Clin North Am. 2014 Apr;28(2):375-86. doi: 10.1016/j.hoc.2013.12.001. Epub 2014 Jan 18.

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

Author information

1
Department of Pediatrics, Hematology-Oncology, Weill Cornell Medical College, 1300 York Avenue, New York, NY 10021, USA. Electronic address: lab2002@med.cornell.edu.
2
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.

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.

KEYWORDS:

ACE-011; ACE-536; Hemoglobinopathies; JAK2; LY-2157299; Steady state; Stress and ineffective erythropoiesis; Transforming growth factor β

PMID:
24589272
PMCID:
PMC3970239
DOI:
10.1016/j.hoc.2013.12.001
[Indexed for MEDLINE]
Free PMC Article

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