2010/05/01 by Derek A. Persons · 1 citation
Biochemistry, Genetics and Molecular Biology · Medicine · #Virus-based gene therapy research #CAR-T cell therapy research #CRISPR and Genetic Engineering
paper · pdf · doi:10.1038/mt.2010.70
openalex publication_date 2010/05/01 · openalex created_date 2025/10/10 · openalex updated_date 2026/07/28
Over the past decade, more than 30 patients with different immunodeficiency disorders have been treated successfully using murine leukemia virus (MLV)-based γ-retroviral vectors to transfer therapeutic genes to autologous hematopoietic cells.1,2,3 However, this approach has been complicated by adverse events caused by γ-retroviral vector integration into the target cell genome. Five cases of lymphoid leukemia have been reported from two different X-linked severe combined immunodeficiency (SCID-X1) trials,4,5 while a recent report now documents two cases of myelodysplasia occurring in a clinical trial for X-linked chronic granulomatous disease.