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A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID

30 Apr 2024

Abstract excerpt

Hematopoietic stem cell gene therapy (GT) using a γ-retroviral vector (γ-RV) is an effective treatment for Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency. Here, we describe a case of GT-related T-cell acute lymphoblastic leukemia (T-ALL) that developed 4.7 years after treatment. The patient underwent chemotherapy and haploidentical transplantation and is currently in remission. Blast cells...

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PMID: 38688902
DOI: 10.1038/s41467-024-47866-5

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