Inclusion of a short hairpin RNA targeting BCL11A into a β-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin
将针对 BCL11A 的短发夹 RNA 纳入 β-珠蛋白表达载体，可以同时合成治疗性成人血红蛋白和胎儿血红蛋白
期刊:Haematologica
影响因子:8.2
doi:10.3324/haematol.2020.276634
Silvia Pires Lourenco, Danuta Jarocha, Valentina Ghiaccio, Amaliris Guerra, Osheiza Abdulmalik, Ping La, Alexandra Zezulin, Kim Smith-Whitley, Janet L Kwiatkowski, Virginia Guzikowski, Yukio Nakamura, Tobias Raabe, Laura Breda, Stefano Rivella
