Research ArticleGene Therapy

Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and coexpressed truncated erythropoietin receptor

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Science Translational Medicine  28 Apr 2021:
Vol. 13, Issue 591, eabb0411
DOI: 10.1126/scitranslmed.abb0411

Article Information

vol. 13 no. 591

PubMed: 
Print ISSN: 
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History: 
  • Received for publication January 25, 2020
  • Resubmitted October 13, 2020
  • Accepted for publication April 2, 2021
  • .

Author Information

  1. Naoya Uchida1,2,*,
  2. Francesca Ferrara3,
  3. Claire M. Drysdale1,
  4. Morgan Yapundich1,
  5. Jackson Gamer1,
  6. Tina Nassehi1,
  7. Julia DiNicola1,
  8. Yoshitaka Shibata1,
  9. Matthew Wielgosz3,
  10. Yoon-Sang Kim3,
  11. Matthew Bauler4,
  12. Robert E. Throm4,
  13. Juan J. Haro-Mora1,
  14. Selami Demirci1,
  15. Aylin C. Bonifacino5,
  16. Allen E. Krouse5,
  17. N. Seth Linde5,
  18. Robert E. Donahue1,
  19. Byoung Ryu3,6 and
  20. John F. Tisdale1
  1. 1Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
  2. 2Division of Molecular and Medical Genetics, Center for Gene and Cell Therapy, The Institute of Medical Science, The University of Tokyo, Minato-ku, Tokyo 108-8639, Japan.
  3. 3Department of Hematology, St. Jude Children’s Research Hospital (SJCRH), Memphis, TN 38105, USA.
  4. 4Vector Development and Production Laboratory, SJCRH, Memphis, TN 38105, USA.
  5. 5Translational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20892, USA.
  6. 6Umoja Biopharma, 1920 Terry Ave., Seattle, WA 98101, USA.
  1. *Corresponding author. Email: uchidan{at}nhlbi.nih.gov

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