Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease
Matthew M. Hsieh, Melissa Bonner, Francis J. Pierciey, Naoya Uchida, James B. Rottman, Laura A. Demopoulos, Manfred Schmidt, Julie Kanter, Mark C. Walters, Alexis A. Thompson, Mohammed Asmal, John F. Tisdale
Abstract
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.
Topics & Concepts
Genetic enhancementMyelodysplastic syndromesMedicineDiseaseAdverse effectOncologyViral vectorVector (molecular biology)BioinformaticsInternal medicineGeneBiologyGeneticsBone marrowRecombinant DNAParvovirus B19 Infection StudiesVirus-based gene therapy researchPrenatal Screening and Diagnostics