Haematologica (May 2021)
Inclusion of a short hairpin RNA targeting <i>BCL11A</i> 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
Affiliations
- Silvia Pires Lourenco
- 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
- Danuta Jarocha
- Department of Pediatrics, Hematology, The Children’s Hospital of Philadelphia, Philadelphia
- Valentina Ghiaccio
- Department of Pediatrics, Hematology, The Children’s Hospital of Philadelphia, Philadelphia
- Amaliris Guerra
- Department of Pediatrics, Hematology, The Children’s Hospital of Philadelphia, Philadelphia
- Osheiza Abdulmalik
- Department of Pediatrics, Hematology, The Children’s Hospital of Philadelphia, Philadelphia
- Ping La
- Department of Pediatrics, Hematology, The Children’s Hospital of Philadelphia, Philadelphia
- Alexandra Zezulin
- Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia
- Kim Smith-Whitley
- Department of Pediatrics, Hematology, The Children’s Hospital of Philadelphia, Philadelphia
- Janet L. Kwiatkowski
- Department of Pediatrics, Hematology, The Children’s Hospital of Philadelphia, Philadelphia
- Virginia Guzikowski
- Department of Pediatrics, Hematology, The Children’s Hospital of Philadelphia, Philadelphia
- Yukio Nakamura
- Cell Engineering Division, RIKEN BioResource Research Center, Tsukuba
- Tobias Raabe
- Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia
- Laura Breda
- Department of Pediatrics, Hematology, The Children’s Hospital of Philadelphia, Philadelphia
- Stefano Rivella
- Department of Pediatrics, Hematology, The Children’s Hospital of Philadelphia, Philadelphia
- DOI
- https://doi.org/10.3324/haematol.2020.276634
- Journal volume & issue
-
Vol. 106,
no. 10
Abstract
No abstracts available.
