Coordinated β-globin expression and α2-globin reduction in a multiplex lentiviral gene therapy vector for β-thalassemia
针对 β-地中海贫血的多重慢病毒基因治疗载体中 β-珠蛋白的协调表达和 α2-珠蛋白的减少
期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2021.04.037
Tiwaporn Nualkaew, Karine Sii-Felice, Marie Giorgi, Bradley McColl, Julie Gouzil, Astrid Glaser, Hsiao P J Voon, Hsin Y Tee, George Grigoriadis, Saovaros Svasti, Suthat Fucharoen, Suradej Hongeng, Philippe Leboulch, Emmanuel Payen, Jim Vadolas2
