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Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease

Hsieh, M. M.; Bonner, M.; Pierciey, F. J.; Uchida, N.; Rottman, J.; Demopoulos, L.; Schmidt, M.; Kanter, J.; Walters, M. C.; Thompson, A. A.; Asmal, M.; Tisdale, J. F.

Blood Adv. 2020 May 13; 4(9):2058-2063

Abstract

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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