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Review
. 2018 Apr;32(2):343-352.
doi: 10.1016/j.hoc.2017.11.002. Epub 2017 Dec 15.

Emerging Therapies

Amaliris Guerra  1 , Khaled M Musallam  2 , Ali T Taher  3 , Stefano Rivella  4
Affiliations
Review

Emerging Therapies

Amaliris Guerra et al. Hematol Oncol Clin North Am. 2018 Apr.

Erratum in

  • Erratum.
    [No authors listed] [No authors listed] Hematol Oncol Clin North Am. 2018 Jun;32(3):xiii. doi: 10.1016/j.hoc.2018.04.009. Hematol Oncol Clin North Am. 2018. PMID: 29729789 No abstract available.

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.

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Conflict of interest statement

Conflict of Interest statement

K.M. received honoraria from Novartis, Celgene, and CRISPR Therapeutics. A.T. received honoraria and research support from Novartis and Celgene. S.R. is a member of scientific advisory board of Ionis Pharmaceuticals. S.R. has grants from Ionis and NIH. A.G. declares no conflict of interest.

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