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Hematol Oncol Clin North Am. 2014 Apr;28(2):199-216. doi: 10.1016/j.hoc.2013.12.003.

Gene therapy for hemoglobinopathies: the state of the field and the future.

Author information

1
Division of Hematology, Oncology and Bone Marrow Transplant, Cancer and Blood Disease Institute (CBDI), Cincinnati Children's Hospital Medical Center (CCHMC), 3333 Burnet Avenue, Cincinnati, OH 45229, USA.
2
Division of Experimental Hematology/Cancer Biology, Cincinnati Children's Research Foundation, Cancer and Blood Institute (CBDI), Cincinnati Children's Hospital Medical Center (CCHMC), 3333 Burnet Avenue, Cincinnati, OH 45229, USA; Division of Hematology, Cincinnati Children's Research Foundation, Cancer and Blood Institute (CBDI), Cincinnati Children's Hospital Medical Center (CCHMC), 3333 Burnet Avenue, Cincinnati, OH 45229, USA. Electronic address: punam.malik@cchmc.org.

Abstract

After nearly two decades of struggle, gene therapy for hemoglobinopathies using vectors carrying β or γ-globin gene has finally reached the clinical doorsteps. This was made possible by advances made in our understanding of critical regulatory elements required for high level of globin gene expression and improved gene transfer vectors and methodologies. Development of gene editing technologies and reprogramming somatic cells for regenerative medicine holds the promise of genetic correction of hemoglobinopathies in the future. This article will review the state of the field and the upcoming technologies that will allow genetic therapeutic correction of hemoglobinopathies.

KEYWORDS:

Gene therapy; Hemoglobinopathy; Sickle cell disease; Thalassemia

PMID:
24589262
PMCID:
PMC4167426
DOI:
10.1016/j.hoc.2013.12.003
[Indexed for MEDLINE]
Free PMC Article

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