Jason Skowronski

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Publications (4)

• Functional screening in human HSPCs identifies optimized protein-based enhancers of Homology Directed Repair
  Nature Communications 2024 DOI: 10.1038/s41467-024-46816-5
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• One Year Follow-up on the First Patient Treated with Nula-Cel: An Autologous CRISPR/Cas9 Gene Corrected CD34+ Cell Product to Treat Sickle Cell Disease
  David C. Shyr, Robert Lowsky, Weston P. Miller +37 more
  Blood 2023 DOI: 10.1182/blood-2023-188963
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• Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease
  Liwen Xu, Premanjali Lahiri, Jason Skowronski

  +3 more

  • Neehar Bhatia
  • Annalisa Lattanzi
  • Matthew Hebden Porteus
  Molecular Therapy - Methods & Clinical Development 2023 DOI: 10.1016/j.omtm.2023.07.009
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• Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease
  Annalisa Lattanzi, Joab Camarena, Premanjali Lahiri +20 more
  Science Translational Medicine 2021 DOI: 10.1126/scitranslmed.abf2444
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