Inclusion of a shRNA targeting BCL11A into a β-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin.

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Authors: 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


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