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Nova · Professor Researcher · re-ranking top 20…

John Fraser Wright

Verified

University of Pennsylvania · Rehabilitation Medicine

Active 1969–2024

h-index73
Citations30.2k
Papers16017 last 5y
Funding
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Research topics

  • Immunology
  • Biology
  • Genetics
  • Cancer research
  • Medicine

Selected publications

  • Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease

    Science Translational Medicine · 2021 · 157 citations

    • Cancer research
    • Medicine
    • Biology

    allelic correction in clinical-scale gcHBB-SCD manufacturing. After transplant into immunodeficient NSG mice, 20% gene correction was achieved with multilineage engraftment. The long-term safety, tumorigenicity, and toxicology study demonstrated no evidence of abnormal hematopoiesis, genotoxicity, or tumorigenicity from the engrafted gcHBB-SCD drug product. Together, these preclinical data support the safety, efficacy, and reproducibility of this gene correction strategy for initiation of a phase 1/2 clinical trial in patients with SCD.

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