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Customizing the genome as therapy for the β-hemoglobinopathies

7 Apr 2016

Abstract excerpt

Despite nearly complete understanding of the genetics of the β-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...

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PMID: 27053533
DOI: 10.1182/blood-2016-01-678128

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