Emerging Therapies

Hematol Oncol Clin North Am. 2018 Apr;32(2):343-352. doi: 10.1016/j.hoc.2017.11.002. Epub 2017 Dec 15.

Abstract

At present, the only definitive cure for β-thalassemia is a bone marrow transplant (BMT); however, HLA-blood-matched donors are scarcely available. Current therapies undergoing clinical investigation with most potential for therapeutic benefit are the β-globin gene transfer of patient-specific hematopoietic stem cells followed by autologous BMT. Other emerging therapies deliver exogenous regulators of several key modulators of erythropoiesis or iron homeostasis. This review focuses on current approaches for the treatment of hemoglobinopathies caused by disruptions of β-globin.

Keywords: Gene transfer; Hemichromes; New therapies; Trap ligands; β-Globin; β-Thalassemia.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Animals
  • Clinical Trials as Topic
  • Combined Modality Therapy / adverse effects
  • Combined Modality Therapy / methods
  • Disease Management
  • Drug Discovery
  • Drug Evaluation, Preclinical
  • Erythropoiesis / genetics
  • Humans
  • beta-Globins / genetics
  • beta-Thalassemia / blood
  • beta-Thalassemia / genetics
  • beta-Thalassemia / therapy*

Substances

  • beta-Globins