Nonclinical evaluation of <i>HBG1/2</i> and <i>BCL11A</i> as genome-editing targets for the treatment of β-hemoglobinopathies
Tusneem Janoudi, Mrunali Jagdale, Meng Wu, Sanjana Gorlla, Peisheng Zhang, Ying Shao, Li Li, Sheryl R. Bowley, Eugenio Marco, Kai‐Hsin Chang
Abstract
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Topics & Concepts
Genome editingGenomeComputational biologyGeneticsMedicineBiologyGeneHemoglobinopathies and Related DisordersCRISPR and Genetic Engineering