Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease

  1. Varun Katta
  2. Kiera O’Keefe
  3. Yichao Li
  4. Thiyagaraj Mayuranathan
  5. Cicera R. Lazzarotto
  6. Rachael K. Wood
  7. Rachel M. Levine
  8. Alicia Powers
  9. Kalin Mayberry
  10. Garret Manquen
  11. Yu Yao
  12. Jingjing Zhang
  13. Yoonjeong Jang
  14. Nikitha Nimmagadda
  15. Erin A. Dempsey
  16. GaHyun Lee
  17. Naoya Uchida
  18. Yong Cheng
  19. Frank Fazio
  20. Tim Lockey
  21. Mike Meagher
  22. Akshay Sharma
  23. John F. Tisdale
  24. Sheng Zhou
  25. Jonathan S. Yen
  26. Mitchell J. Weiss
  27. Shengdar Q. Tsai
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  1. Version published to 10.1016/j.ymthe.2024.07.022
  2. Version published to 10.1101/2024.04.30.591737 on bioRxiv