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A pause in gene therapy: Reflecting on the unique challenges of sickle cell disease

20 Mar 2021

Abstract excerpt

Recently, the largest lentiviral vector (LV)-mediated β-globin replacement gene therapy trial in sickle cell disease (SCD) was suspended due to a reported suspected unexpected serious adverse reaction (SUSAR) of acute myeloid leukemia (AML).1 Over the last 6 years, 47 patients with SCD have been treated with LV gene therapy in two related clinical trials (ClinicalTrials.gov: NCT02140554 and NCT04293185) that have...

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PMID: 33743192
DOI: 10.1016/j.ymthe.2021.03.010

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