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

Blood Adv. 2020 May 12;4(9):2058-2063. doi: 10.1182/bloodadvances.2019001330.


  1. Ability to accurately attribute adverse events post–gene therapy is required to describe the benefit-risk of these novel treatments.

  2. A SCD patient developed myelodysplastic syndrome post-LentiGlobin treatment; we show how insertional oncogenesis was excluded as the cause.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Anemia, Sickle Cell* / genetics
  • Anemia, Sickle Cell* / therapy
  • Genetic Therapy
  • Humans
  • Lentivirus / genetics
  • Myelodysplastic Syndromes* / genetics
  • Myelodysplastic Syndromes* / therapy