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

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

Haematologica（2021）

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