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

Emerging Therapies.

Author information

1
Department of Pediatrics, Division of Hematology, Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA.
2
International Network of Hematology, 31-33 High Holborn, London WC1V 6AX, UK.
3
Department of Internal Medicine, American University of Beirut Medical Center, PO Box: 11-0236, Cairo Street, Hamra, Raid E Solh, Beirut 1107 2020, Lebanon.
4
Department of Pediatrics, Division of Hematology, Children's Hospital of Philadelphia (CHOP), Philadelphia, PA, USA; Cell and Molecular Biology Graduate Group (CAMB), University of Pennsylvania, 421 Curie Boulevard/6064 160 Biomedical Research Building (BRB) 2/3, Philadelphia, PA 19104-6064, USA. Electronic address: rivellas@email.chop.edu.

Erratum in

  • Erratum. [Hematol Oncol Clin North Am. 2018]

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

PMID:
29458736
PMCID:
PMC5823282
[Available on 2019-04-01]
DOI:
10.1016/j.hoc.2017.11.002
[Indexed for MEDLINE]

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