Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells.
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R. Majeti | R. Bak | G. Rettig | M. Behlke | Christopher A. Vakulskas | Michael A. Collingwood | M. Porteus | Yankai Zhang | V. Sheehan | J. Tisdale | D. Dever | J. Camarena | Nicole M. Bode | Benjamin J. Lesch | N. Uchida | Renata M. Martin | W. Srifa | M. Cromer | Viktor T Lemgart | Ankush Goyal | Feifei Zhao | Ezequiel Ponce | Gavin L. Kurgan