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Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease
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https://doi.org/10.1182/bloodadvances.2019001330Abstract
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.
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