American Society of Hematology, Blood Advances, 9(4), p. 2058-2063, 2020
DOI: 10.1182/bloodadvances.2019001330
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Key Points Ability to accurately attribute adverse events post–gene therapy is required to describe the benefit-risk of these novel treatments. A SCD patient developed myelodysplastic syndrome post-LentiGlobin treatment; we show how insertional oncogenesis was excluded as the cause.