https://www.selleckchem.com/
Recently, encouraging data provided long-awaited hope for gene therapy as a cure for sickle cell disease (SCD). Nevertheless, the suspension of the bluebird bio gene therapy trial (ClinicalTrials.gov NCT02140554) after participants developed acute myeloid leukemia/myelodysplastic syndrome (AML/MDS) is concerning. Potential possibilities for these cases include busulfan, insertional mutagenesis, both or neither. Busulfan was considered the cause in the first reported case, as the transgene was not present in the AML/MDS. However, busulfan is unlikely to have
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