Research ArticleHEALTH AND MEDICINE

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

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Science Advances  12 Feb 2020:
Vol. 6, no. 7, eaay9392
DOI: 10.1126/sciadv.aay9392

Article Information

vol. 6 no. 7

PubMed: 
Online ISSN: 
History: 
  • Received for publication July 30, 2019
  • Accepted for publication November 25, 2019
  • .

Author Information

  1. Leslie Weber1,2,3,*,
  2. Giacomo Frati3,4,*,
  3. Tristan Felix3,4,
  4. Giulia Hardouin3,4,
  5. Antonio Casini5,
  6. Clara Wollenschlaeger3,4,
  7. Vasco Meneghini3,4,
  8. Cecile Masson6,
  9. Anne De Cian7,
  10. Anne Chalumeau1,3,4,
  11. Fulvio Mavilio8,9,
  12. Mario Amendola10,
  13. Isabelle Andre-Schmutz1,4,
  14. Anna Cereseto5,
  15. Wassim El Nemer11,12,13,
  16. Jean-Paul Concordet7,
  17. Carine Giovannangeli7,
  18. Marina Cavazzana1,4,14 and
  19. Annarita Miccio3,4,
  1. 1Laboratory of Human Lymphohematopoiesis, INSERM UMR1163, Paris, France.
  2. 2Paris Diderot University–Sorbonne Paris Cité, Paris, France.
  3. 3Laboratory of chromatin and gene regulation during development, INSERM UMR1163, Paris, France.
  4. 4Paris Descartes–Sorbonne Paris Cité University, Imagine Institute, Paris, France.
  5. 5CIBIO, University of Trento, Trento, Italy.
  6. 6Paris-Descartes Bioinformatics Platform, Imagine Institute, Paris 75015, France.
  7. 7INSERM U1154, CNRS UMR7196, Museum National d’Histoire Naturelle, Paris, France.
  8. 8Department of Life Sciences, University of Modena and Reggio Emilia, Modena, Italy.
  9. 9Audentes Therapeutics, San Francisco, CA, USA.
  10. 10Genethon, INSERM UMR951, Evry, France.
  11. 11Biologie Intégrée du Globule Rouge UMR_S1134, Inserm, Univ. Paris Diderot, Sorbonne Paris Cité, Univ. de la Réunion, Univ. des Antilles, Paris, France.
  12. 12Institut National de la Transfusion Sanguine, F-75015 Paris, France.
  13. 13Laboratoire d’Excellence GR-Ex, Paris, France.
  14. 14Biotherapy Department, Necker Children’s Hospital, Assistance Publique–Hôpitaux de Paris, Paris, France.
  1. Corresponding author. Email: annarita.miccio{at}institutimagine.org
  • * These authors contributed equally to this work as co-first authors.

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