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

Haematologica. 2021 Oct 1;106(10):2740-2745. doi: 10.3324/haematol.2020.276634.

¹ Department of Pediatrics, Hematology, The Children's Hospital of Philadelphia, Philadelphia; Graduate Program in Basic and Applied Biology (GABBA), Institute of Biomedical Sciences Abel Salazar, University of Porto, Porto.
² Department of Pediatrics, Hematology, The Children's Hospital of Philadelphia, Philadelphia. [email protected].
³ Department of Pediatrics, Hematology, The Children's Hospital of Philadelphia, Philadelphia.
⁴ Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia.
⁵ Cell Engineering Division, RIKEN BioResource Research Center, Tsukuba.

No abstract available

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