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

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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Detalhes bibliográficos
Publicado no:Blood Adv
Main Authors: Hsieh, Matthew M., Bonner, Melissa, Pierciey, Francis John, Uchida, Naoya, Rottman, James, Demopoulos, Laura, Schmidt, Manfred, Kanter, Julie, Walters, Mark C., Thompson, Alexis A., Asmal, Mohammed, Tisdale, John F.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2020
Assuntos:
Stimulus Report
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7218414/
https://ncbi.nlm.nih.gov/pubmed/32396618
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2019001330
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