Customizing the genome as therapy for the β-hemoglobinopathies.
作者： Matthew C. Canver , Stuart H. Orkin
DOI: 10.1182/BLOOD-2016-01-678128
关键词:
摘要: Despite nearly complete understanding of the genetics β-hemoglobinopathies for several decades, definitive treatment options have lagged behind. Recent developments in technologies facile manipulation genome (zinc finger nucleases, transcription activator-like effector or clustered regularly interspaced short palindromic repeats–based nucleases) raise prospects their clinical application. The use genome-editing autologous CD34+ hematopoietic stem and progenitor cells represents a promising therapeutic avenue β-globin disorders. Genetic correction strategies relying on homology-directed repair pathway may genetic defects, whereas disruption nonhomologous end joining induce compensatory fetal hemoglobin expression. Harnessing power editing usher second-generation form gene therapy
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