Journal
BLOOD
Volume 127, Issue 21, Pages 2536-2545Publisher
AMER SOC HEMATOLOGY
DOI: 10.1182/blood-2016-01-678128
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Funding
- National Institutes of Health (NIH) National Institute of Diabetes and Digestive and Kidney Diseases [F30DK 103359]
- NIH National Heart, Lung, and Blood Institute [HL032259, P01HL032262]
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Despite nearly complete understanding of the genetics of the beta-hemoglobinopathies for several decades, definitive treatment options have lagged behind. Recent developments in technologies for facile manipulation of the genome (zinc finger nucleases, transcription activator-like effector nucleases, or clustered regularly interspaced short palindromic repeats-based nucleases) raise prospects for their clinical application. The use of genome-editing technologies in autologous CD34(+) hematopoietic stem and progenitor cells represents a promising therapeutic avenue for the beta-globin disorders. Genetic correction strategies relying on the homology-directed repair pathway may repair genetic defects, whereas genetic disruption strategies relying on the nonhomologous end joining pathway may induce compensatory fetal hemoglobin expression. Harnessing the power of genome editing may usher in a second-generation form of gene therapy for the b-globin disorders.
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