Inclusion of a short hairpin RNA targeting BCL11A into a b-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin

por Lourenco, Silvia Pires, Jarocha, Danuta, Ghiaccio, Valentina, Guerra, Amaliris, Abdulmalik, Osheiza, La, Ping, Zezulin, Alexandra, Smith-Whitley, Kim, Kwiatkowski, Janet L., Guzikowski, Virginia, Nakamura, Yukio, Raabe, Tobias, Breda, Laura, Rivella, Stefano
Publicado 2021