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Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither

3 Jun 2021

Abstract excerpt

Recently, encouraging data provided long-awaited hope for gene therapy as a cure for sickle cell disease (SCD). Nevertheless, the transient suspension of the bluebird bio gene therapy trial (clinicaltrials.gov: NCT02140554) after participants developed acute myeloid leukemia/myelodysplastic syndrome (AML/MDS) raised concerns. Potential possibilities for these cases include busulfan, insertional mutagenesis, both,...

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PMID: 34115136
DOI: 10.1182/blood.2021011488

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