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Inclusion of a short hairpin RNA targeting BCL11A into a ß-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin.
Pires Lourenco, Silvia; 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.
Afiliação
  • Pires Lourenco S; 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.
  • Jarocha D; Department of Pediatrics, Hematology, The Children's Hospital of Philadelphia, Philadelphia. jarochad@email.chop.edu.
  • Ghiaccio V; Department of Pediatrics, Hematology, The Children's Hospital of Philadelphia, Philadelphia.
  • Guerra A; Department of Pediatrics, Hematology, The Children's Hospital of Philadelphia, Philadelphia.
  • Abdulmalik O; Department of Pediatrics, Hematology, The Children's Hospital of Philadelphia, Philadelphia.
  • La P; Department of Pediatrics, Hematology, The Children's Hospital of Philadelphia, Philadelphia.
  • Zezulin A; Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia.
  • Smith-Whitley K; Department of Pediatrics, Hematology, The Children's Hospital of Philadelphia, Philadelphia.
  • Kwiatkowski JL; Department of Pediatrics, Hematology, The Children's Hospital of Philadelphia, Philadelphia.
  • Guzikowski V; Department of Pediatrics, Hematology, The Children's Hospital of Philadelphia, Philadelphia.
  • Nakamura Y; Cell Engineering Division, RIKEN BioResource Research Center, Tsukuba.
  • Raabe T; Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia.
  • Breda L; Department of Pediatrics, Hematology, The Children's Hospital of Philadelphia, Philadelphia.
  • Rivella S; Department of Pediatrics, Hematology, The Children's Hospital of Philadelphia, Philadelphia.
Haematologica ; 106(10): 2740-2745, 2021 10 01.
Article em En | MEDLINE | ID: mdl-34047176
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Hemoglobina Fetal / Gama-Globinas Limite: Adult / Humans Idioma: En Revista: Haematologica Ano de publicação: 2021 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Hemoglobina Fetal / Gama-Globinas Limite: Adult / Humans Idioma: En Revista: Haematologica Ano de publicação: 2021 Tipo de documento: Article